{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T16:28:27Z","timestamp":1790958507985,"version":"4.1.0"},"reference-count":344,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2020,9,1]],"date-time":"2020-09-01T00:00:00Z","timestamp":1598918400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2020,9,1]],"date-time":"2020-09-01T00:00:00Z","timestamp":1598918400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2020,6,26]],"date-time":"2020-06-26T00:00:00Z","timestamp":1593129600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100016036","name":"Health~Holland","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100016036","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100016062","name":"Stichting Metakids","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100016062","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100016065","name":"Stichting Stofwisselkracht","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100016065","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2020,9]]},"DOI":"10.1016\/j.omtm.2020.06.022","type":"journal-article","created":{"date-parts":[[2020,7,3]],"date-time":"2020-07-03T20:45:48Z","timestamp":1593809148000},"page":"532-557","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":92,"special_numbering":"C","title":["Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease"],"prefix":"10.1016","volume":"18","author":[{"given":"Martijn P.T.","family":"Ernst","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mike","family":"Broeders","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pablo","family":"Herrero-Hernandez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Esmee","family":"Oussoren","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ans T.","family":"van der Ploeg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"W.W.M. Pim","family":"Pijnappel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2020.06.022_bib1","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.omtm.2017.11.007","article-title":"Emerging issues in AAV-mediated in\u00a0vivo gene therapy","volume":"8","author":"Colella","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2020.06.022_bib2","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1038\/nature15818","article-title":"Gene therapy returns to centre stage","volume":"526","author":"Naldini","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib3","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1016\/j.ymthe.2020.01.001","article-title":"Immune responses to viral gene therapy vectors","volume":"28","author":"Shirley","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib4","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1023\/B:NEON.0000003652.71665.f2","article-title":"Clinical trials with retrovirus mediated gene therapy\u2014what have we learned?","volume":"65","author":"Rainov","year":"2003","journal-title":"J.\u00a0Neurooncol."},{"key":"10.1016\/j.omtm.2020.06.022_bib5","doi-asserted-by":"crossref","first-page":"670","DOI":"10.3389\/fimmu.2020.00670","article-title":"Human immune responses to adeno-associated virus (AAV) vectors","volume":"11","author":"Ronzitti","year":"2020","journal-title":"Front. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib6","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1016\/j.tibtech.2013.04.004","article-title":"ZFN, TALEN, and CRISPR\/Cas-based methods for genome engineering","volume":"31","author":"Gaj","year":"2013","journal-title":"Trends Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib7","doi-asserted-by":"crossref","first-page":"100789","DOI":"10.1016\/j.isci.2019.100789","article-title":"Sharpening the molecular scissors: advances in gene-editing technology","volume":"23","author":"Broeders","year":"2020","journal-title":"iScience"},{"key":"10.1016\/j.omtm.2020.06.022_bib8","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib9","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A\u2022T to G\u2022C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib10","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1038\/nrc3179","article-title":"Safe harbours for the integration of new DNA in the human genome","volume":"12","author":"Sadelain","year":"2011","journal-title":"Nat. Rev. Cancer"},{"key":"10.1016\/j.omtm.2020.06.022_bib11","doi-asserted-by":"crossref","first-page":"1975","DOI":"10.1016\/j.stemcr.2018.04.002","article-title":"Large-scale expansion of human iPSC-derived skeletal muscle cells for disease modeling and cell-based therapeutic strategies","volume":"10","author":"van der Wal","year":"2018","journal-title":"Stem Cell Reports"},{"key":"10.1016\/j.omtm.2020.06.022_bib12","doi-asserted-by":"crossref","first-page":"827","DOI":"10.1038\/nbt.2647","article-title":"DNA targeting specificity of RNA-guided Cas9 nucleases","volume":"31","author":"Hsu","year":"2013","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib13","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1038\/nbt.4192","article-title":"Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements","volume":"36","author":"Kosicki","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib14","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1038\/nm.4313","article-title":"Refining strategies to translate genome editing to the clinic","volume":"23","author":"Cornu","year":"2017","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib15","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1146\/annurev-biochem-013118-111730","article-title":"Evaluating and enhancing target specificity of gene-editing nucleases and deaminases","volume":"88","author":"Kim","year":"2019","journal-title":"Annu. Rev. Biochem."},{"key":"10.1016\/j.omtm.2020.06.022_bib16","first-page":"1902312","article-title":"CRISPR\/Cas systems in genome editing: methodologies and tools for sgRNA design, off-target evaluation, and strategies to mitigate off-target effects","volume":"7","author":"Manghwar","year":"2020","journal-title":"Adv. Sci. (Weinh.)"},{"key":"10.1016\/j.omtm.2020.06.022_bib17","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/B978-0-12-801185-0.00003-9","article-title":"Determining the specificities of TALENs, Cas9, and other genome-editing enzymes","volume":"546","author":"Pattanayak","year":"2014","journal-title":"Methods Enzymol."},{"key":"10.1016\/j.omtm.2020.06.022_bib18","doi-asserted-by":"crossref","first-page":"387","DOI":"10.1038\/nrd.2016.280","article-title":"Delivery technologies for genome editing","volume":"16","author":"Yin","year":"2017","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtm.2020.06.022_bib19","doi-asserted-by":"crossref","first-page":"1234","DOI":"10.1080\/10717544.2018.1474964","article-title":"Delivering CRISPR: a review of the challenges and approaches","volume":"25","author":"Lino","year":"2018","journal-title":"Drug Deliv."},{"key":"10.1016\/j.omtm.2020.06.022_bib20","doi-asserted-by":"crossref","first-page":"726","DOI":"10.1038\/s41578-019-0145-9","article-title":"Engineered materials for in\u00a0vivo delivery of genome-editing machinery","volume":"4","author":"Tong","year":"2019","journal-title":"Nat. Rev. Mater."},{"key":"10.1016\/j.omtm.2020.06.022_bib21","doi-asserted-by":"crossref","first-page":"1432","DOI":"10.1016\/j.ymthe.2020.04.017","article-title":"AAV-CRISPR gene editing is negated by pre-existing immunity to Cas9","volume":"28","author":"Li","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib22","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1038\/s41591-018-0326-x","article-title":"Identification of preexisting adaptive immunity to Cas9 proteins in humans","volume":"25","author":"Charlesworth","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib23","doi-asserted-by":"crossref","first-page":"3497","DOI":"10.1038\/s41467-018-05843-9","article-title":"Cas9 immunity creates challenges for CRISPR gene editing therapies","volume":"9","author":"Crudele","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2020.06.022_bib24","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/s41580-019-0131-5","article-title":"The next generation of CRISPR-Cas technologies and applications","volume":"20","author":"Pickar-Oliver","year":"2019","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.omtm.2020.06.022_bib25","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/s12276-019-0339-7","article-title":"Recent advances in the CRISPR genome editing tool set","volume":"51","author":"Moon","year":"2019","journal-title":"Exp. Mol. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib26","first-page":"653","article-title":"Genome editing: past, present, and future","volume":"90","author":"Carroll","year":"2017","journal-title":"Yale J. Biol. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib27","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1038\/s10038-017-0352-4","article-title":"In\u00a0vivo genome editing via the HITI method as a tool for gene therapy","volume":"63","author":"Suzuki","year":"2018","journal-title":"J.\u00a0Hum. Genet."},{"key":"10.1016\/j.omtm.2020.06.022_bib28","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1038\/nrm.2017.48","article-title":"Non-homologous DNA end joining and alternative pathways to double-strand break repair","volume":"18","author":"Chang","year":"2017","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.omtm.2020.06.022_bib29","doi-asserted-by":"crossref","first-page":"1121","DOI":"10.1016\/j.tibtech.2019.03.008","article-title":"CRISPR\/Cas-mediated base editing: technical considerations and practical applications","volume":"37","author":"Molla","year":"2019","journal-title":"Trends Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib30","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib31","doi-asserted-by":"crossref","first-page":"569","DOI":"10.1038\/nbt.2908","article-title":"Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing","volume":"32","author":"Tsai","year":"2014","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib32","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/nature16526","article-title":"High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects","volume":"529","author":"Kleinstiver","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib33","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1126\/science.aad5227","article-title":"Rationally engineered Cas9 nucleases with improved specificity","volume":"351","author":"Slaymaker","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.06.022_bib34","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1038\/nbt.4066","article-title":"A highly specific SpCas9 variant is identified by in\u00a0vivo screening in yeast","volume":"36","author":"Casini","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib35","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1038\/nature24268","article-title":"Enhanced proofreading governs CRISPR-Cas9 targeting accuracy","volume":"550","author":"Chen","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib36","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1038\/nature14592","article-title":"Engineered CRISPR-Cas9 nucleases with altered PAM specificities","volume":"523","author":"Kleinstiver","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib37","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1038\/nbt.2808","article-title":"Improving CRISPR-Cas nuclease specificity using truncated guide RNAs","volume":"32","author":"Fu","year":"2014","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib38","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1038\/s41587-019-0095-1","article-title":"Increasing the specificity of CRISPR systems with engineered RNA secondary structures","volume":"37","author":"Kocak","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib39","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1038\/nchembio.2559","article-title":"Partial DNA-guided Cas9 enables genome editing with reduced off-target activity","volume":"14","author":"Yin","year":"2018","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.omtm.2020.06.022_bib40","doi-asserted-by":"crossref","first-page":"107501","DOI":"10.1016\/j.pharmthera.2020.107501","article-title":"Recent advances in genome editing of stem cells for drug discovery and therapeutic application","volume":"209","author":"Lee","year":"2020","journal-title":"Pharmacol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib41","doi-asserted-by":"crossref","first-page":"359","DOI":"10.1016\/j.omtm.2019.02.008","article-title":"Advancements and obstacles of CRISPR-Cas9 technology in translational research","volume":"13","author":"You","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2020.06.022_bib42","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1146\/annurev-med-112717-094629","article-title":"Ethics of human genome editing","volume":"70","author":"Coller","year":"2019","journal-title":"Annu. Rev. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib43","doi-asserted-by":"crossref","first-page":"1479","DOI":"10.1038\/s41556-019-0424-0","article-title":"Human germline genome editing","volume":"21","author":"Lea","year":"2019","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.omtm.2020.06.022_bib44","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1016\/j.ajhg.2017.06.012","article-title":"Human germline genome editing","volume":"101","author":"Ormond","year":"2017","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.omtm.2020.06.022_bib45","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.critrevonc.2019.01.015","article-title":"Unleash the power of the mighty T\u00a0cells-basis of adoptive cellular therapy","volume":"136","author":"Sukari","year":"2019","journal-title":"Crit. Rev. Oncol. Hematol."},{"key":"10.1016\/j.omtm.2020.06.022_bib46","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1097\/PPO.0000000000000158","article-title":"Endogenous T-cell therapy: clinical experience","volume":"21","author":"Yee","year":"2015","journal-title":"Cancer J."},{"key":"10.1016\/j.omtm.2020.06.022_bib47","doi-asserted-by":"crossref","first-page":"646","DOI":"10.1016\/j.cell.2011.02.013","article-title":"Hallmarks of cancer: the next generation","volume":"144","author":"Hanahan","year":"2011","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2020.06.022_bib48","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1038\/nrc3239","article-title":"The blockade of immune checkpoints in cancer immunotherapy","volume":"12","author":"Pardoll","year":"2012","journal-title":"Nat. Rev. Cancer"},{"key":"10.1016\/j.omtm.2020.06.022_bib49","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1016\/j.intimp.2018.06.001","article-title":"Immune checkpoint blockade therapy for cancer: an overview of FDA-approved immune checkpoint inhibitors","volume":"62","author":"Hargadon","year":"2018","journal-title":"Int. Immunopharmacol."},{"key":"10.1016\/j.omtm.2020.06.022_bib50","doi-asserted-by":"crossref","first-page":"2443","DOI":"10.1056\/NEJMoa1200690","article-title":"Safety, activity, and immune correlates of anti-PD-1 antibody in cancer","volume":"366","author":"Topalian","year":"2012","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib51","doi-asserted-by":"crossref","first-page":"2455","DOI":"10.1056\/NEJMoa1200694","article-title":"Safety and activity of anti-PD-L1 antibody in patients with advanced cancer","volume":"366","author":"Brahmer","year":"2012","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib52","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1056\/NEJMoa1305133","article-title":"Safety and tumor responses with lambrolizumab (anti-PD-1) in melanoma","volume":"369","author":"Hamid","year":"2013","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib53","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1016\/j.ejca.2015.11.016","article-title":"Immune-related adverse events with immune checkpoint blockade: a comprehensive review","volume":"54","author":"Michot","year":"2016","journal-title":"Eur. J. Cancer"},{"key":"10.1016\/j.omtm.2020.06.022_bib54","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1016\/j.jim.2005.11.023","article-title":"In\u00a0vitro methods for generating CD8+ T-cell clones for immunotherapy from the na\u00efve repertoire","volume":"310","author":"Ho","year":"2006","journal-title":"J.\u00a0Immunol. Methods"},{"key":"10.1016\/j.omtm.2020.06.022_bib55","doi-asserted-by":"crossref","first-page":"1380","DOI":"10.1038\/mt.2015.71","article-title":"Clinical scale zinc finger nuclease-mediated gene editing of PD-1 in tumor infiltrating lymphocytes for the treatment of metastatic melanoma","volume":"23","author":"Beane","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib56","doi-asserted-by":"crossref","first-page":"20070","DOI":"10.1038\/srep20070","article-title":"CRISPR-Cas9 mediated efficient PD-1 disruption on human primary T\u00a0cells from cancer patients","volume":"6","author":"Su","year":"2016","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib57","doi-asserted-by":"crossref","first-page":"e000311","DOI":"10.1136\/jitc-2019-000311","article-title":"Increased antitumor efficacy of PD-1-deficient melanoma-specific human lymphocytes","volume":"8","author":"Marotte","year":"2020","journal-title":"J.\u00a0Immunother. Cancer"},{"key":"10.1016\/j.omtm.2020.06.022_bib58","doi-asserted-by":"crossref","first-page":"2087","DOI":"10.1158\/0008-5472.CAN-15-3352","article-title":"TALEN-mediated inactivation of PD-1 in tumor-reactive lymphocytes promotes intratumoral T-cell persistence and rejection of established tumors","volume":"76","author":"Menger","year":"2016","journal-title":"Cancer Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib59","doi-asserted-by":"crossref","first-page":"5208","DOI":"10.18632\/oncotarget.23730","article-title":"CRISPR knock out of programmed cell death protein 1 enhances anti-tumor activity of cytotoxic T lymphocytes","volume":"9","author":"Zhao","year":"2017","journal-title":"Oncotarget"},{"key":"10.1016\/j.omtm.2020.06.022_bib60","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1166\/jbn.2019.2712","article-title":"Generation of cancer-specific cytotoxic PD-1\u2212 T\u00a0cells using liposome-encapsulated CRISPR\/Cas system with dendritic\/tumor fusion cells","volume":"15","author":"Lu","year":"2019","journal-title":"J.\u00a0Biomed. Nanotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib61","unstructured":"Wu, S.; Hangzhou Cancer Hospital, Ltd.; Anhui Kedgene Biotechnology Co., Ltd. (2017). PD-1 knockout engineered T\u00a0cells for advanced esophageal cancer. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03081715."},{"key":"10.1016\/j.omtm.2020.06.022_bib62","unstructured":"Lu, Y.; Sichuan University; Chengdu MedGenCell, Co., Ltd. (2016). PD-1 knockout engineered T\u00a0cells for metastatic non-small cell lung cancer. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02793856."},{"key":"10.1016\/j.omtm.2020.06.022_bib63","unstructured":"Chen, S.; Guangzhou Anjie Biomedical Technology Co., Ltd.; University of Technology, Sydney (2018). Therapeutic vaccine plus PD-1 knockout in prostate cancer treatment. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03525652."},{"key":"10.1016\/j.omtm.2020.06.022_bib64","unstructured":"Yang, Y.; The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School (2017). PD-1 knockout EBV-CTLs for advanced stage Epstein-Barr virus (EBV) associated malignancies. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03044743."},{"key":"10.1016\/j.omtm.2020.06.022_bib65","doi-asserted-by":"crossref","first-page":"732","DOI":"10.1038\/s41591-020-0840-5","article-title":"Safety and feasibility of CRISPR-edited T\u00a0cells in patients with refractory non-small-cell lung cancer","volume":"26","author":"Lu","year":"2020","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib66","doi-asserted-by":"crossref","first-page":"e000695","DOI":"10.1136\/jitc-2020-000695","article-title":"Lifting the innate immune barriers to antitumor immunity","volume":"8","author":"Rothlin","year":"2020","journal-title":"J.\u00a0Immunother. Cancer"},{"key":"10.1016\/j.omtm.2020.06.022_bib67","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1038\/s41577-018-0061-z","article-title":"Natural killer cells and other innate lymphoid cells in cancer","volume":"18","author":"Chiossone","year":"2018","journal-title":"Nat. Rev. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib68","article-title":"Efficient genome editing of human natural killer cells by CRISPR RNP","author":"Rautela","year":"2018","journal-title":"bioRxiv"},{"key":"10.1016\/j.omtm.2020.06.022_bib69","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1016\/j.ymthe.2019.10.009","article-title":"A genetically engineered primary human natural killer cell platform for cancer immunotherapy","volume":"28","author":"Pomeroy","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib70","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1111\/imr.12136","article-title":"Adoptive immunotherapy for cancer","volume":"257","author":"Ruella","year":"2014","journal-title":"Immunol. Rev."},{"key":"10.1016\/j.omtm.2020.06.022_bib71","first-page":"39","article-title":"CRISPR\/Cas9 genome editing: fueling the revolution in cancer immunotherapy","volume":"66","author":"Liu","year":"2018","journal-title":"Curr. Res. Transl. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib72","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1038\/nrc3565","article-title":"Gene-engineered T\u00a0cells for cancer therapy","volume":"13","author":"Kershaw","year":"2013","journal-title":"Nat. Rev. Cancer"},{"key":"10.1016\/j.omtm.2020.06.022_bib73","first-page":"956304","article-title":"Chimeric antigen receptor-engineered T\u00a0cells for immunotherapy of cancer","volume":"2010","author":"Cartellieri","year":"2010","journal-title":"J.\u00a0Biomed. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib74","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1093\/intimm\/dxy006","article-title":"Generation of tumor antigen-specific murine CD8+ T\u00a0cells with enhanced anti-tumor activity via highly efficient CRISPR\/Cas9 genome editing","volume":"30","author":"Ouchi","year":"2018","journal-title":"Int. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib75","doi-asserted-by":"crossref","first-page":"1118","DOI":"10.3389\/fphar.2018.01118","article-title":"Disruption of PD-1 enhanced the anti-tumor activity of chimeric antigen receptor T\u00a0cells against hepatocellular carcinoma","volume":"9","author":"Guo","year":"2018","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.omtm.2020.06.022_bib76","doi-asserted-by":"crossref","first-page":"446","DOI":"10.1089\/hum.2017.234","article-title":"Nucleofection with plasmid DNA for CRISPR\/Cas9-mediated inactivation of programmed cell death protein 1 in CD133-specific CAR T\u00a0cells","volume":"30","author":"Hu","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib77","doi-asserted-by":"crossref","first-page":"365","DOI":"10.1007\/s00262-018-2281-2","article-title":"CRISPR\/Cas9-mediated PD-1 disruption enhances human mesothelin-targeted CAR T\u00a0cell effector functions","volume":"68","author":"Hu","year":"2019","journal-title":"Cancer Immunol. Immunother."},{"key":"10.1016\/j.omtm.2020.06.022_bib78","doi-asserted-by":"crossref","first-page":"737","DOI":"10.1038\/s41598-017-00462-8","article-title":"CRISPR\/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T\u00a0cells","volume":"7","author":"Rupp","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib79","doi-asserted-by":"crossref","DOI":"10.1007\/s12253-019-00759-1","article-title":"EGFRvIII-CAR-T cells with PD-1 knockout have improved anti-glioma activity","author":"Zhu","year":"2020","journal-title":"Pathol. Oncol. Res."},{"issue":"Suppl","key":"10.1016\/j.omtm.2020.06.022_bib80","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1200\/JCO.2017.35.15_suppl.103","article-title":"The effect of pembrolizumab in combination with CD19-targeted chimeric antigen receptor (CAR) T\u00a0cells in relapsed acute lymphoblastic leukemia (ALL)","volume":"35","author":"Maude","year":"2017","journal-title":"J.\u00a0Clin. Oncol."},{"key":"10.1016\/j.omtm.2020.06.022_bib81","doi-asserted-by":"crossref","first-page":"1039","DOI":"10.1182\/blood-2016-09-738245","article-title":"PD-1 blockade modulates chimeric antigen receptor (CAR)-modified T\u00a0cells: refueling the CAR","volume":"129","author":"Chong","year":"2017","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib82","unstructured":"Shang, X.; Third Military Medical University (2017). CD19 CAR and PD-1 knockout engineered T\u00a0cells for CD19 positive malignant B-cell derived leukemia and lymphoma. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03298828."},{"key":"10.1016\/j.omtm.2020.06.022_bib83","unstructured":"Weidong, H.; Chinese PLA General Hospital (2018). Study of PD-1 gene-knocked out mesothelin-directed CAR-T cells with the conditioning of PC in mesothelin positive multiple solid tumors. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03747965."},{"key":"10.1016\/j.omtm.2020.06.022_bib84","unstructured":"Chen, S.; Guangzhou Anjie Biomedical Technology Co., Ltd. (2018). CAR T and PD-1 knockout engineered T\u00a0cells for esophageal cancer. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03706326."},{"key":"10.1016\/j.omtm.2020.06.022_bib85","unstructured":"Chen, S.; Guangzhou Anjie Biomedical Technology Co., Ltd.; University of Technology, Sydney (2018). Anti-MUC1 CAR T\u00a0cells and PD-1 knockout engineered T\u00a0cells for NSCLC. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03525782."},{"key":"10.1016\/j.omtm.2020.06.022_bib86","unstructured":"Guangxun, G.; Xi\u2019An Yufan Biotechnology Co., Ltd. (2019). CRISPR (HPK1) edited CD19-specific CAR-T cells (XYF19 CAR-T cells) for CD19+ leukemia or lymphoma. https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT04037566."},{"key":"10.1016\/j.omtm.2020.06.022_bib87","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1038\/ni1416","article-title":"Hematopoietic progenitor kinase 1 negatively regulates T\u00a0cell receptor signaling and T\u00a0cell-mediated immune responses","volume":"8","author":"Shui","year":"2007","journal-title":"Nat. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib88","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1007\/s00262-009-0761-0","article-title":"Hematopoietic progenitor kinase 1 is a critical component of prostaglandin E2-mediated suppression of the anti-tumor immune response","volume":"59","author":"Alzabin","year":"2010","journal-title":"Cancer Immunol. Immunother."},{"key":"10.1016\/j.omtm.2020.06.022_bib89","doi-asserted-by":"crossref","first-page":"e0212670","DOI":"10.1371\/journal.pone.0212670","article-title":"Critical role of kinase activity of hematopoietic progenitor kinase 1 in anti-tumor immune surveillance","volume":"14","author":"Liu","year":"2019","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2020.06.022_bib90","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1016\/j.celrep.2018.09.012","article-title":"The kinase activity of hematopoietic progenitor kinase 1 is essential for the regulation of T\u00a0cell function","volume":"25","author":"Hernandez","year":"2018","journal-title":"Cell Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib91","doi-asserted-by":"crossref","first-page":"911","DOI":"10.1007\/s00280-019-03800-x","article-title":"Disruption of CTLA-4 expression on peripheral blood CD8\u00a0+ T\u00a0cell enhances anti-tumor efficacy in bladder cancer","volume":"83","author":"Zhang","year":"2019","journal-title":"Cancer Chemother. Pharmacol."},{"key":"10.1016\/j.omtm.2020.06.022_bib92","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1016\/j.gene.2017.09.010","article-title":"CRISPR knock out CTLA-4 enhances the anti-tumor activity of cytotoxic T lymphocytes","volume":"636","author":"Shi","year":"2017","journal-title":"Gene"},{"key":"10.1016\/j.omtm.2020.06.022_bib93","doi-asserted-by":"crossref","first-page":"4692","DOI":"10.1158\/0008-5472.CAN-18-0030","article-title":"CRISPR\/Cas9-mediated knockout of DGK improves antitumor activities of human T\u00a0cells","volume":"78","author":"Jung","year":"2018","journal-title":"Cancer Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib94","doi-asserted-by":"crossref","first-page":"882","DOI":"10.1038\/ni958","article-title":"Enhanced T\u00a0cell responses due to diacylglycerol kinase \u03b6 deficiency","volume":"4","author":"Zhong","year":"2003","journal-title":"Nat. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib95","doi-asserted-by":"crossref","first-page":"554","DOI":"10.1007\/s11684-017-0543-6","article-title":"CRISPR-Cas9 mediated LAG-3 disruption in CAR-T cells","volume":"11","author":"Zhang","year":"2017","journal-title":"Front. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib96","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1007\/s11899-017-0417-7","article-title":"Genome-editing technologies in adoptive T\u00a0cell immunotherapy for cancer","volume":"12","author":"Singh","year":"2017","journal-title":"Curr. Hematol. Malig. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib97","doi-asserted-by":"crossref","first-page":"1178","DOI":"10.1038\/mt.2016.106","article-title":"Translational implications for off-the-shelf immune cells expressing chimeric antigen receptors","volume":"24","author":"Torikai","year":"2016","journal-title":"mol. ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib98","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1097\/MOH.0000000000000181","article-title":"Challenges and opportunities of allogeneic donor-derived CAR T\u00a0cells","volume":"22","author":"Yang","year":"2015","journal-title":"Curr. Opin. Hematol."},{"key":"10.1016\/j.omtm.2020.06.022_bib99","doi-asserted-by":"crossref","first-page":"5697","DOI":"10.1182\/blood-2012-01-405365","article-title":"A foundation for universal T-cell based immunotherapy: T\u00a0cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR","volume":"119","author":"Torikai","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib100","doi-asserted-by":"crossref","first-page":"3853","DOI":"10.1158\/0008-5472.CAN-14-3321","article-title":"Multiplex genome-edited T-cell manufacturing platform for \u201coff-the-shelf\u201d adoptive T-cell immunotherapies","volume":"75","author":"Poirot","year":"2015","journal-title":"Cancer Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib101","doi-asserted-by":"crossref","first-page":"1215","DOI":"10.1016\/j.ymthe.2018.02.025","article-title":"Long terminal repeat CRISPR-CAR-coupled \u201cuniversal\u201d T\u00a0cells mediate potent anti-leukemic effects","volume":"26","author":"Georgiadis","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib102","doi-asserted-by":"crossref","first-page":"eaaj2013","DOI":"10.1126\/scitranslmed.aaj2013","article-title":"Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T\u00a0cells","volume":"9","author":"Qasim","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib103","unstructured":"Servier (Institut de Recherches Internationales Servier); ADIR, a Servier Group company (2016). Study of UCART19 in pediatric patients with relapsed\/refractory b acute lymphoblastic leukemia. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02808442."},{"key":"10.1016\/j.omtm.2020.06.022_bib104","unstructured":"Servier (Institut de Recherches Internationales Servier); ADIR, a Servier Group company (2016). Dose escalation study of UCART19 in adult patients with relapsed refractory B-cell acute lymphoblastic leukaemia. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02746952."},{"key":"10.1016\/j.omtm.2020.06.022_bib105","unstructured":"Allogene Therapeutics (2019). Safety and efficacy of ALLO-501 Anti-CD19 allogeneic CAR T\u00a0cells in adults with relapsed\/refractory large B cell or follicular lymphoma (ALPHA). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03939026."},{"key":"10.1016\/j.omtm.2020.06.022_bib106","unstructured":"Allogene Therapeutics (2019). Safety and efficacy of ALLO-715 BCMA allogenic CAR T\u00a0cells in in adults with relapsed or refractory multiple myeloma (UNIVERSAL). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04093596."},{"key":"10.1016\/j.omtm.2020.06.022_bib107","unstructured":"Servier (Institut de Recherches Internationales Servier); ADIR, a Servier Group company (2016). A study to evaluate the long-term safety of patients with advanced lymphoid malignancies who have been previously administered with UCART19\/ALLO-501. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02735083."},{"key":"10.1016\/j.omtm.2020.06.022_bib108","doi-asserted-by":"crossref","first-page":"17002","DOI":"10.18632\/oncotarget.15218","article-title":"A versatile system for rapid multiplex genome-edited CAR T\u00a0cell generation","volume":"8","author":"Ren","year":"2017","journal-title":"Oncotarget"},{"key":"10.1016\/j.omtm.2020.06.022_bib109","doi-asserted-by":"crossref","first-page":"2255","DOI":"10.1158\/1078-0432.CCR-16-1300","article-title":"Multiplex genome editing to generate universal CAR T\u00a0cells resistant to PD1 inhibition","volume":"23","author":"Ren","year":"2017","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib110","doi-asserted-by":"crossref","first-page":"154","DOI":"10.1038\/cr.2016.142","article-title":"CRISPR-Cas9-mediated multiplex gene editing in CAR-T cells","volume":"27","author":"Liu","year":"2017","journal-title":"Cell Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib111","doi-asserted-by":"crossref","first-page":"304","DOI":"10.1186\/s40425-019-0806-7","article-title":"CRISPR-Cas9 disruption of PD-1 enhances activity of universal EGFRvIII CAR T\u00a0cells in a preclinical model of human glioblastoma","volume":"7","author":"Choi","year":"2019","journal-title":"J.\u00a0Immunother. Cancer"},{"key":"10.1016\/j.omtm.2020.06.022_bib112","unstructured":"Weidong, H.; Chinese PLA General Hospital (2017). A study evaluating UCART019 in patients with relapsed or refractory CD19+ leukemia and lymphoma. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03166878."},{"key":"10.1016\/j.omtm.2020.06.022_bib113","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1038\/nature21405","article-title":"Targeting a CAR to the TRAC locus with CRISPR\/Cas9 enhances tumour rejection","volume":"543","author":"Eyquem","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib114","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1038\/s41586-018-0326-5","article-title":"Reprogramming human T\u00a0cell function and specificity with non-viral genome targeting","volume":"559","author":"Roth","year":"2018","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib115","unstructured":"CRISPR Therapeutics AG (2019). A safety and efficacy study evaluating CTX110 in subjects with relapsed or refractory B-cell malignancies. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04035434."},{"key":"10.1016\/j.omtm.2020.06.022_bib116","unstructured":"CRISPR Therapeutics AG (2020). A safety and efficacy study evaluating CTX120 in subjects with relapsed or refractory multiple myeloma. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04244656."},{"key":"10.1016\/j.omtm.2020.06.022_bib117","unstructured":"Xu, K.L., and Zheng, J.N.; Nanjing Bioheng Biotech Co., Ltd. (2019). CTA101\u00a0UCAR-T cell injection for treatment of relapsed or refractory CD19+ B-cell acute lymphoblastic leukemia. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04154709."},{"key":"10.1016\/j.omtm.2020.06.022_bib118","unstructured":"Cellectis S.A. (2019). Phase I study of UCART22 in patients with relapsed or refractory CD22+ B-cell acute lymphoblastic leukemia (BALLI-01). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04150497."},{"key":"10.1016\/j.omtm.2020.06.022_bib119","unstructured":"The First Affiliated Hospital with Nanjing Medical University; Nanjing Bioheng Biotech Co., Ltd. (2019). CTA101 in the treatment of relapsed or refractory diffuse large B-cell lymphoma. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04026100."},{"key":"10.1016\/j.omtm.2020.06.022_bib120","unstructured":"Weidong, H.; Chinese PLA General Hospital (2018). A feasibility and safety study of universal dual specificity CD19 and CD20 or CD22 CAR-T cell immunotherapy for relapsed or refractory leukemia and lymphoma. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03398967."},{"key":"10.1016\/j.omtm.2020.06.022_bib121","unstructured":"Cellectis S.A. (2017). Study evaluating safety and efficacy of UCART123 in patients with relapsed\/ refractory acute myeloid leukemia. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03190278."},{"key":"10.1016\/j.omtm.2020.06.022_bib122","unstructured":"Cellectis S.A. (2019). Study evaluating safety and efficacy of UCART targeting CS1 in patients with relapsed\/refractory multiple myeloma (MELANI-01). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04142619."},{"key":"10.1016\/j.omtm.2020.06.022_bib123","unstructured":"Zhang, X.; Gracell Biotechnologies (Shanghai) Co., Ltd.; 920th Hospital of Joint Logistics Support Force; The Second Affiliated Hospital of Chongqing Medical University; The Affiliated Hospital of Guizhou Medical University; Central South University; The First Affiliated Hospital of Kunming Medical College; The General Hospital of Western Theater Command; Second Affiliated Hospital of Xi\u2019an Jiaotong University; Nanfang Hospital of Southern Medical University; Fujian Medical University Union Hospital; The First Affiliated Hospital of Anhui Medical University; Tang-Du Hospital (2020). Anti-CD19\u00a0U-CAR-T cell therapy for B cell hematologic malignancies. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04264039."},{"key":"10.1016\/j.omtm.2020.06.022_bib124","unstructured":"Xiang, X.; Gracell Biotechnologies (Shanghai) Co., Ltd.; 920th Hospital of Joint Logistics Support Force; The Second Affiliated Hospital of Chongqing Medical University; The Affiliated Hospital of Guizhou Medical University; Central South University; The First Affiliated Hospital of Kunming Medical College; The General Hospital of Western Theater Command; Second Affiliated Hospital of Xi\u2019an Jiaotong University; Nanfang Hospital of Southern Medical University; Fujian Medical University Union Hospital; The First Affiliated Hospital of Anhui Medical University; Tang-Du Hospital (2020). Anti-CD7\u00a0U-CAR-T cell therapy for T\/NK cell hematologic malignancies. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04264078."},{"key":"10.1016\/j.omtm.2020.06.022_bib125","unstructured":"Shanghai Bioray Laboratory Inc.; Shanghai Tongji Hospital, Tongji University School of Medicine; Second Xiangya Hospital of Central South University (2018). Efficacy and safety evaluation of BCMA-UCART. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03752541."},{"key":"10.1016\/j.omtm.2020.06.022_bib126","unstructured":"Shanghai Bioray Laboratory Inc.; The First Affiliated Hospital of Zhengzhou University; Second Xiangya Hospital of Central South University; Shanghai 10th People\u2019s Hospital (2017). Safety and efficacy evaluation of CD19-UCART. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03229876."},{"key":"10.1016\/j.omtm.2020.06.022_bib127","unstructured":"Shanghai Longyao Biotechnology Inc. Ltd. (2019). The clinical study of CD19\u00a0UCAR-T cells in patients with B-cell acute lymphoblastic leukemia (B-ALL). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04166838."},{"key":"10.1016\/j.omtm.2020.06.022_bib128","doi-asserted-by":"crossref","first-page":"1370","DOI":"10.1080\/10428194.2018.1553300","article-title":"Adoptive cell therapy for acute myeloid leukemia","volume":"60","author":"Ma","year":"2019","journal-title":"Leuk. Lymphoma"},{"key":"10.1016\/j.omtm.2020.06.022_bib129","doi-asserted-by":"crossref","first-page":"1637","DOI":"10.1038\/leu.2015.52","article-title":"CD33-specific chimeric antigen receptor T\u00a0cells exhibit potent preclinical activity against human acute myeloid leukemia","volume":"29","author":"Kenderian","year":"2015","journal-title":"Leukemia"},{"key":"10.1016\/j.omtm.2020.06.022_bib130","doi-asserted-by":"crossref","first-page":"2343","DOI":"10.1182\/blood-2013-09-529537","article-title":"Preclinical targeting of human acute myeloid leukemia and myeloablation using chimeric antigen receptor-modified T\u00a0cells","volume":"123","author":"Gill","year":"2014","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib131","doi-asserted-by":"crossref","first-page":"1439","DOI":"10.1016\/j.cell.2018.05.013","article-title":"Genetic inactivation of CD33 in hematopoietic stem cells to enable CAR T\u00a0cell immunotherapy for acute myeloid leukemia","volume":"173","author":"Kim","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2020.06.022_bib132","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.omtm.2017.01.005","article-title":"Genome-wide specificity of highly efficient TALENs and CRISPR\/Cas9 for T\u00a0cell receptor modification","volume":"4","author":"Knipping","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2020.06.022_bib133","unstructured":"University of Pennsylvania (2018). NY-ESO-1-redirected CRISPR (TCRendo and PD1) edited T\u00a0cells (NYCE T\u00a0cells). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03399448."},{"key":"10.1016\/j.omtm.2020.06.022_bib134","unstructured":"Weidong, H.; Chinese PLA General Hospital (2018). Study of CRISPR-Cas9 mediated PD-1 and TCR gene-knocked out mesothelin-directed CAR-T cells in patients with mesothelin positive multiple solid tumors. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03545815."},{"key":"10.1016\/j.omtm.2020.06.022_bib135","doi-asserted-by":"crossref","first-page":"eaba7365","DOI":"10.1126\/science.aba7365","article-title":"CRISPR-engineered T\u00a0cells in patients with refractory cancer","volume":"367","author":"Stadtmauer","year":"2020","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.06.022_bib136","doi-asserted-by":"crossref","first-page":"914","DOI":"10.1038\/nm.3910","article-title":"NY-ESO-1-specific TCR-engineered T\u00a0cells mediate sustained antigen-specific antitumor effects in myeloma","volume":"21","author":"Rapoport","year":"2015","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib137","doi-asserted-by":"crossref","first-page":"1019","DOI":"10.1158\/1078-0432.CCR-14-2708","article-title":"A pilot trial using lymphocytes genetically engineered with an NY-ESO-1-reactive T-cell receptor: long-term follow-up and correlates with response","volume":"21","author":"Robbins","year":"2015","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib138","doi-asserted-by":"crossref","first-page":"944","DOI":"10.1158\/2159-8290.CD-17-1417","article-title":"Antitumor activity associated with prolonged persistence of adoptively transferred NY-ESO-1 c259T cells in synovial sarcoma","volume":"8","author":"D\u2019Angelo","year":"2018","journal-title":"Cancer Discov."},{"key":"10.1016\/j.omtm.2020.06.022_bib139","doi-asserted-by":"crossref","first-page":"2307","DOI":"10.1038\/s41375-018-0285-8","article-title":"CAR T-cells for T-cell malignancies: challenges in distinguishing between therapeutic, normal, and neoplastic T-cells","volume":"32","author":"Alcantara","year":"2018","journal-title":"Leukemia"},{"key":"10.1016\/j.omtm.2020.06.022_bib140","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1080\/08923973.2019.1637889","article-title":"T cells expressing CD26-specific chimeric antigen receptors exhibit extensive self-antigen-driven fratricide","volume":"41","author":"Zhou","year":"2019","journal-title":"Immunopharmacol. Immunotoxicol."},{"key":"10.1016\/j.omtm.2020.06.022_bib141","doi-asserted-by":"crossref","first-page":"3869","DOI":"10.1172\/JCI43437","article-title":"MHC-restricted fratricide of human lymphocytes expressing survivin-specific transgenic T\u00a0cell receptors","volume":"120","author":"Leisegang","year":"2010","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2020.06.022_bib142","doi-asserted-by":"crossref","first-page":"1970","DOI":"10.1038\/s41375-018-0065-5","article-title":"An \u201coff-the-shelf\u201d fratricide-resistant CAR-T for the treatment of T\u00a0cell hematologic malignancies","volume":"32","author":"Cooper","year":"2018","journal-title":"Leukemia"},{"key":"10.1016\/j.omtm.2020.06.022_bib143","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1182\/blood-2017-01-761320","article-title":"CD7-edited T\u00a0cells expressing a CD7-specific CAR for the therapy of T-cell malignancies","volume":"130","author":"Gomes-Silva","year":"2017","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib144","unstructured":"Rouce, R.; The Methodist Hospital System; Center for Cell and Gene Therapy, Baylor College of Medicine (2018). Cell therapy for high risk T-cell malignancies using CD7-specific CAR expressed on autologous T\u00a0cells. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03690011."},{"key":"10.1016\/j.omtm.2020.06.022_bib145","first-page":"69","article-title":"Global cancer statistics","volume":"61","author":"Jemal","year":"2011","journal-title":"CA Cancer J. Clin."},{"key":"10.1016\/j.omtm.2020.06.022_bib146","doi-asserted-by":"crossref","first-page":"e453","DOI":"10.1016\/S2214-109X(16)30099-7","article-title":"Global estimates of human papillomavirus vaccination coverage by region and income level: a pooled analysis","volume":"4","author":"Bruni","year":"2016","journal-title":"Lancet Glob. Health"},{"key":"10.1016\/j.omtm.2020.06.022_bib147","doi-asserted-by":"crossref","first-page":"889","DOI":"10.1016\/S0140-6736(13)60022-7","article-title":"Human papillomavirus and cervical cancer","volume":"382","author":"Crosbie","year":"2013","journal-title":"Lancet"},{"key":"10.1016\/j.omtm.2020.06.022_bib148","unstructured":"Howlader, N., Noone, A.M., Krapcho, M., Miller, D., Bishop, K., Kosary, C.L., Yu, M., Ruhl, J., Tatalovich, Z., Mariotto, A., Lewis, D.R., Chen, H.S., Feuer, E.J., and Cronin, K.A., eds. (2018). SEER Cancer Statistics Review, 1975\u20132014 (National Cancer Institute), https:\/\/seer.cancer.gov\/csr\/1975_2014\/."},{"key":"10.1016\/j.omtm.2020.06.022_bib149","doi-asserted-by":"crossref","first-page":"342","DOI":"10.1038\/nrc798","article-title":"Papillomaviruses and cancer: from basic studies to clinical application","volume":"2","author":"zur Hausen","year":"2002","journal-title":"Nat. Rev. Cancer"},{"key":"10.1016\/j.omtm.2020.06.022_bib150","doi-asserted-by":"crossref","first-page":"273","DOI":"10.1007\/s10495-007-0163-8","article-title":"RNA interference against HPV16 E7 oncogene leads to viral E6 and E7 suppression in cervical cancer cells and apoptosis via upregulation of Rb and p53","volume":"13","author":"Sima","year":"2008","journal-title":"Apoptosis"},{"key":"10.1016\/j.omtm.2020.06.022_bib151","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1016\/j.canlet.2009.09.022","article-title":"Selective targeting of HPV-16 E6\/E7 in cervical cancer cells with a potent oncolytic adenovirus and its enhanced effect with radiotherapy in\u00a0vitro and vivo","volume":"291","author":"Wang","year":"2010","journal-title":"Cancer Lett."},{"key":"10.1016\/j.omtm.2020.06.022_bib152","doi-asserted-by":"crossref","first-page":"2044","DOI":"10.1016\/j.drudis.2019.07.011","article-title":"Cervical cancer and HPV infection: ongoing therapeutic research to counteract the action of E6 and E7 oncoproteins","volume":"24","author":"Almeida","year":"2019","journal-title":"Drug Discov. Today"},{"key":"10.1016\/j.omtm.2020.06.022_bib153","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1196\/annals.1359.019","article-title":"Development of resistance to RNAi in mammalian cells","volume":"1058","author":"Zheng","year":"2005","journal-title":"Ann. N Y Acad. Sci."},{"key":"10.1016\/j.omtm.2020.06.022_bib154","doi-asserted-by":"crossref","first-page":"6495","DOI":"10.1158\/1078-0432.CCR-14-0250","article-title":"Zinc finger nucleases targeting the human papillomavirus E7 oncogene induce E7 disruption and a transformed phenotype in HPV16\/18-positive cervical cancer cells","volume":"20","author":"Ding","year":"2014","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib155","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1172\/JCI78206","article-title":"TALEN-mediated targeting of HPV oncogenes ameliorates HPV-related cervical malignancy","volume":"125","author":"Hu","year":"2015","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2020.06.022_bib156","doi-asserted-by":"crossref","first-page":"5500","DOI":"10.1038\/s41598-017-05696-0","article-title":"TALEN based HPV-E7 editing triggers necrotic cell death in cervical cancer cells","volume":"7","author":"Shankar","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib157","doi-asserted-by":"crossref","first-page":"1422","DOI":"10.1016\/j.bbrc.2014.07.014","article-title":"In\u00a0vitro and in\u00a0vivo growth suppression of human papillomavirus 16-positive cervical cancer cells by CRISPR\/Cas9","volume":"450","author":"Zhen","year":"2014","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.omtm.2020.06.022_bib158","doi-asserted-by":"crossref","first-page":"612823","DOI":"10.1155\/2014\/612823","article-title":"Disruption of HPV16-E7 by CRISPR\/Cas system induces apoptosis and growth inhibition in HPV16 positive human cervical cancer cells","volume":"2014","author":"Hu","year":"2014","journal-title":"BioMed Res. Int."},{"key":"10.1016\/j.omtm.2020.06.022_bib159","doi-asserted-by":"crossref","first-page":"11965","DOI":"10.1128\/JVI.01879-14","article-title":"Inactivation of the human papillomavirus E6 or E7 gene in cervical carcinoma cells by using a bacterial CRISPR\/Cas RNA-guided endonuclease","volume":"88","author":"Kennedy","year":"2014","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2020.06.022_bib160","unstructured":"Ma, D.; Huazhong University of Science and Technology (2017). Study of molecular-targeted therapy using zinc finger nuclease in cervical precancerous lesions. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02800369."},{"key":"10.1016\/j.omtm.2020.06.022_bib161","unstructured":"Ma, D.; Huazhong University of Science and Technology (2017). Study of targeted therapy using transcription activator-like effector nucleases in cervical precancerous lesions. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03226470."},{"key":"10.1016\/j.omtm.2020.06.022_bib162","unstructured":"Zheng, H.; First Affiliated Hospital, Sun Yat-Sen University; Jingchu University of Technology (2017). A safety and efficacy study of TALEN and CRISPR\/Cas9 in the treatment of HPV-related cervical intraepithelial neoplasia. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03057912."},{"key":"10.1016\/j.omtm.2020.06.022_bib163","doi-asserted-by":"crossref","first-page":"498","DOI":"10.1016\/j.tranon.2016.10.002","article-title":"In\u00a0vitro and in\u00a0vivo synergistic therapeutic effect of cisplatin with human papillomavirus16 E6\/E7 CRISPR\/Cas9 on cervical cancer cell line","volume":"9","author":"Zhen","year":"2016","journal-title":"Transl. Oncol."},{"key":"10.1016\/j.omtm.2020.06.022_bib164","doi-asserted-by":"crossref","first-page":"d641","DOI":"10.2741\/A800","article-title":"The role of human papillomaviruses in human cancers","volume":"7","author":"M\u00fcnger","year":"2002","journal-title":"Front. Biosci."},{"key":"10.1016\/j.omtm.2020.06.022_bib165","doi-asserted-by":"crossref","first-page":"e186","DOI":"10.1038\/mtna.2014.38","article-title":"The CRISPR\/Cas9 system facilitates clearance of the intrahepatic HBV templates in\u00a0vivo","volume":"3","author":"Lin","year":"2014","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2020.06.022_bib166","doi-asserted-by":"crossref","first-page":"e216","DOI":"10.1038\/mtna.2014.68","article-title":"Targeting hepatitis B virus with CRISPR\/Cas9","volume":"3","author":"Seeger","year":"2014","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2020.06.022_bib167","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.virol.2015.02.024","article-title":"Bacterial CRISPR\/Cas DNA endonucleases: a revolutionary technology that could dramatically impact viral research and treatment","volume":"479\u2013480","author":"Kennedy","year":"2015","journal-title":"Virology"},{"key":"10.1016\/j.omtm.2020.06.022_bib168","doi-asserted-by":"crossref","first-page":"404","DOI":"10.1038\/gt.2015.2","article-title":"Harnessing the clustered regularly interspaced short palindromic repeat (CRISPR)\/CRISPR-associated Cas9 system to disrupt the hepatitis B virus","volume":"22","author":"Zhen","year":"2015","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib169","doi-asserted-by":"crossref","first-page":"91","DOI":"10.3389\/fcimb.2017.00091","article-title":"Removal of integrated hepatitis B virus DNA using CRISPR-Cas9","volume":"7","author":"Li","year":"2017","journal-title":"Front. Cell. Infect. Microbiol."},{"key":"10.1016\/j.omtm.2020.06.022_bib170","doi-asserted-by":"crossref","first-page":"7401","DOI":"10.1038\/s41598-017-07642-6","article-title":"ssAAVs containing cassettes encoding SaCas9 and guides targeting hepatitis B virus inactivate replication of the virus in cultured cells","volume":"7","author":"Scott","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib171","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1016\/j.antiviral.2018.02.011","article-title":"Inhibition of hepatitis B virus replication via HBV DNA cleavage by Cas9 from Staphylococcus aureus","volume":"152","author":"Liu","year":"2018","journal-title":"Antiviral Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib172","doi-asserted-by":"crossref","first-page":"440","DOI":"10.1038\/cr.2017.16","article-title":"A non-viral CRISPR\/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in\u00a0vivo","volume":"27","author":"Jiang","year":"2017","journal-title":"Cell Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib173","doi-asserted-by":"crossref","first-page":"13157","DOI":"10.1073\/pnas.1410785111","article-title":"RNA-guided endonuclease provides a therapeutic strategy to cure latent herpesviridae infection","volume":"111","author":"Wang","year":"2014","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2020.06.022_bib174","doi-asserted-by":"crossref","first-page":"626","DOI":"10.1099\/jgv.0.000012","article-title":"CRISPR\/Cas9-mediated genome editing of Epstein-Barr virus in human cells","volume":"96","author":"Yuen","year":"2015","journal-title":"J.\u00a0Gen. Virol."},{"key":"10.1016\/j.omtm.2020.06.022_bib175","doi-asserted-by":"crossref","first-page":"e1005701","DOI":"10.1371\/journal.ppat.1005701","article-title":"CRISPR\/Cas9-mediated genome editing of herpesviruses limits productive and latent infections","volume":"12","author":"van Diemen","year":"2016","journal-title":"PLoS Pathog."},{"key":"10.1016\/j.omtm.2020.06.022_bib176","doi-asserted-by":"crossref","first-page":"296","DOI":"10.1016\/j.virusres.2017.04.019","article-title":"Suppression of Epstein-Barr virus DNA load in latently infected nasopharyngeal carcinoma cells by CRISPR\/Cas9","volume":"244","author":"Yuen","year":"2018","journal-title":"Virus Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib177","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1016\/j.coi.2018.06.002","article-title":"Harnessing CRISPR to combat human viral infections","volume":"54","author":"de Buhr","year":"2018","journal-title":"Curr. Opin. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib178","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1146\/annurev-virology-101416-041646","article-title":"The molecular basis for human immunodeficiency virus latency","volume":"4","author":"Mbonye","year":"2017","journal-title":"Annu. Rev. Virol."},{"key":"10.1016\/j.omtm.2020.06.022_bib179","doi-asserted-by":"crossref","first-page":"874","DOI":"10.1038\/44755","article-title":"Re-emergence of HIV after stopping therapy","volume":"401","author":"Chun","year":"1999","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib180","doi-asserted-by":"crossref","first-page":"831","DOI":"10.3389\/fphar.2017.00831","article-title":"Improving the adherence to antiretroviral therapy, a difficult but essential task for a successful HIV treatment-clinical points of view and practical considerations","volume":"8","author":"Iacob","year":"2017","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.omtm.2020.06.022_bib181","doi-asserted-by":"crossref","first-page":"2791","DOI":"10.1182\/blood-2010-09-309591","article-title":"Evidence for the cure of HIV infection by CCR5\u039432\/\u039432 stem cell transplantation","volume":"117","author":"Allers","year":"2011","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib182","doi-asserted-by":"crossref","first-page":"692","DOI":"10.1056\/NEJMoa0802905","article-title":"Long-term control of HIV by CCR5 delta32\/delta32 stem-cell transplantation","volume":"360","author":"H\u00fctter","year":"2009","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib183","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1038\/384179a0","article-title":"CD4-induced interaction of primary HIV-1 gp120 glycoproteins with the chemokine receptor CCR-5","volume":"384","author":"Wu","year":"1996","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib184","doi-asserted-by":"crossref","first-page":"244","DOI":"10.1038\/s41586-019-1027-4","article-title":"HIV-1 remission following CCR5\u039432\/\u039432 haematopoietic stem-cell transplantation","volume":"568","author":"Gupta","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib185","doi-asserted-by":"crossref","first-page":"782","DOI":"10.7326\/0003-4819-135-9-200111060-00008","article-title":"Effects of CCR5-\u039432, CCR2-64I, and SDF-1 3\u2032A alleles on HIV-1 disease progression: an international meta-analysis of individual-patient data","volume":"135","author":"Ioannidis","year":"2001","journal-title":"Ann. Intern. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib186","doi-asserted-by":"crossref","first-page":"1631","DOI":"10.1097\/00002030-200307250-00007","article-title":"Effects of CCR5-delta32 and CCR2-64I alleles on disease progression of perinatally HIV-1-infected children: an international meta-analysis","volume":"17","author":"Ioannidis","year":"2003","journal-title":"AIDS"},{"key":"10.1016\/j.omtm.2020.06.022_bib187","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1097\/00002030-200302140-00012","article-title":"Effects of CCR5-delta32 and CCR2-64I alleles on HIV-1 disease progression: the protection varies with duration of infection","volume":"17","author":"Mulherin","year":"2003","journal-title":"AIDS"},{"key":"10.1016\/j.omtm.2020.06.022_bib188","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1038\/nbt1410","article-title":"Establishment of HIV-1 resistance in CD4+ T\u00a0cells by genome editing using zinc-finger nucleases","volume":"26","author":"Perez","year":"2008","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib189","unstructured":"University of Pennsylvania; Sangamo Therapeutics (2009). Autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728 for HIV (zinc-finger). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT00842634."},{"key":"10.1016\/j.omtm.2020.06.022_bib190","unstructured":"Sangamo Therapeutics (2010). Phase 1 dose escalation study of autologous t-cells genetically modified at the CCR5 gene by zinc finger nucleases in HIV-infected patients. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT01044654."},{"key":"10.1016\/j.omtm.2020.06.022_bib191","unstructured":"Sangamo Therapeutics (2014). Repeat doses of SB-728mR-T after cyclophosphamide conditioning in HIV-infected subjects on HAART. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02225665."},{"key":"10.1016\/j.omtm.2020.06.022_bib192","unstructured":"University of Pennsylvania; National Institute of Allergy and Infectious Diseases (NIAID) (2015). A phase I study of T-cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728mR in HIV-infected patients. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02388594."},{"key":"10.1016\/j.omtm.2020.06.022_bib193","unstructured":"Sangamo Therapeutics (2012). Dose escalation study of cyclophosphamide in HIV-infected subjects on HAART receiving SB-728-T. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT01543152."},{"key":"10.1016\/j.omtm.2020.06.022_bib194","unstructured":"Smith, C.; Case Western Reserve Univeristy; University of California, San Francisco; University of Cincinnati (2018). CCR5-modified CD4+ T\u00a0cells for HIV infection (TRAILBLAZER). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03666871."},{"key":"10.1016\/j.omtm.2020.06.022_bib195","unstructured":"Sangamo Therapeutics (2014). Study of autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases in HIV-infected subjects. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT01252641."},{"key":"10.1016\/j.omtm.2020.06.022_bib196","doi-asserted-by":"crossref","first-page":"901","DOI":"10.1056\/NEJMoa1300662","article-title":"Gene editing of CCR5 in autologous CD4 T\u00a0cells of persons infected with HIV","volume":"370","author":"Tebas","year":"2014","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib197","unstructured":"Sangamo Therapeutics (2019). Long-term follow-up of HIV subjects exposed to SB-728-T or SB-728mR-T. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04201782."},{"key":"10.1016\/j.omtm.2020.06.022_bib198","doi-asserted-by":"crossref","first-page":"539","DOI":"10.1089\/apc.2016.0233","article-title":"Clinical applications of genome editing to HIV cure","volume":"30","author":"Wang","year":"2016","journal-title":"AIDS Patient Care STDS"},{"key":"10.1016\/j.omtm.2020.06.022_bib199","doi-asserted-by":"crossref","first-page":"2546","DOI":"10.1182\/blood-2016-01-678144","article-title":"The clinical applications of genome editing in HIV","volume":"127","author":"Wang","year":"2016","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib200","doi-asserted-by":"crossref","first-page":"1259","DOI":"10.1038\/mt.2013.65","article-title":"Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases","volume":"21","author":"Li","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib201","doi-asserted-by":"crossref","first-page":"1256","DOI":"10.1038\/nbt.3408","article-title":"Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors","volume":"33","author":"Wang","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib202","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1038\/nbt.1663","article-title":"Human hematopoietic stem\/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in\u00a0vivo","volume":"28","author":"Holt","year":"2010","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib203","unstructured":"Krishnan, A.Y.; City of Hope Medical Center; Sangamo Therapeutics;California Institute for Regenerative Medicine (CIRM) (2015). Safety study of zinc finger nuclease CCR5-modified hematopoietic stem\/progenitor cells in HIV-1 infected patients. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02500849."},{"key":"10.1016\/j.omtm.2020.06.022_bib204","unstructured":"Hu, C.; Affiliated Hospital to Academy of Military Medical Sciences;Peking University; Capital Medical University (2017). Safety of transplantation of CRISPR CCR5 modified CD34+ cells in HIV-infected subjects with hematological malignances. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03164135."},{"key":"10.1016\/j.omtm.2020.06.022_bib205","doi-asserted-by":"crossref","first-page":"605","DOI":"10.1038\/s41577-018-0042-2","article-title":"CAR T\u00a0cells for infection, autoimmunity and allotransplantation","volume":"18","author":"Maldini","year":"2018","journal-title":"Nat. Rev. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib206","unstructured":"Tebas, P; University of Pennsylvania (2018). CD4 CAR+ ZFN-modified T\u00a0cells in HIV therapy. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03617198."},{"key":"10.1016\/j.omtm.2020.06.022_bib207","doi-asserted-by":"crossref","first-page":"e2747","DOI":"10.1371\/journal.pone.0002747","article-title":"CCR5\u039432 genotypes in a German HIV-1 seroconverter cohort and report of HIV-1 infection in a CCR5\u039432 homozygous individual","volume":"3","author":"Oh","year":"2008","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2020.06.022_bib208","doi-asserted-by":"crossref","first-page":"880","DOI":"10.1056\/NEJMc1405805","article-title":"Shift of HIV tropism in stem-cell transplantation with CCR5 Delta32 mutation","volume":"371","author":"Kordelas","year":"2014","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib209","doi-asserted-by":"crossref","first-page":"867","DOI":"10.1097\/QAD.0000000000000629","article-title":"Viremic control and viral coreceptor usage in two HIV-1-infected persons homozygous for CCR5 \u039432","volume":"29","author":"Henrich","year":"2015","journal-title":"AIDS"},{"key":"10.1016\/j.omtm.2020.06.022_bib210","doi-asserted-by":"crossref","first-page":"407","DOI":"10.4049\/jimmunol.1600343","article-title":"The latent reservoir for HIV-1: how immunologic memory and clonal expansion contribute to HIV-1 persistence","volume":"197","author":"Murray","year":"2016","journal-title":"J.\u00a0Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib211","doi-asserted-by":"crossref","first-page":"2510","DOI":"10.1038\/srep02510","article-title":"Harnessing the CRISPR\/Cas9 system to disrupt latent HIV-1 provirus","volume":"3","author":"Ebina","year":"2013","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib212","doi-asserted-by":"crossref","first-page":"11461","DOI":"10.1073\/pnas.1405186111","article-title":"RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection","volume":"111","author":"Hu","year":"2014","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2020.06.022_bib213","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1186\/s12977-015-0150-z","article-title":"The CRISPR\/Cas9 system inactivates latent HIV-1 proviral DNA","volume":"12","author":"Zhu","year":"2015","journal-title":"Retrovirology"},{"key":"10.1016\/j.omtm.2020.06.022_bib214","doi-asserted-by":"crossref","first-page":"22555","DOI":"10.1038\/srep22555","article-title":"Elimination of HIV-1 genomes from human T-lymphoid cells by CRISPR\/Cas9 gene editing","volume":"6","author":"Kaminski","year":"2016","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib215","doi-asserted-by":"crossref","first-page":"2819","DOI":"10.1016\/j.celrep.2016.11.057","article-title":"A combinatorial CRISPR-Cas9 attack on HIV-1 DNA extinguishes all infectious provirus in infected T\u00a0cell cultures","volume":"17","author":"Wang","year":"2016","journal-title":"Cell Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib216","doi-asserted-by":"crossref","first-page":"6413","DOI":"10.1038\/ncomms7413","article-title":"Use of the CRISPR\/Cas9 system as an intracellular defense against HIV-1 infection in human cells","volume":"6","author":"Liao","year":"2015","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2020.06.022_bib217","doi-asserted-by":"crossref","first-page":"1163","DOI":"10.1097\/QAD.0000000000001079","article-title":"Functional screening of guide RNAs targeting the regulatory and structural HIV-1 viral genome for a cure of AIDS","volume":"30","author":"Yin","year":"2016","journal-title":"AIDS"},{"key":"10.1016\/j.omtm.2020.06.022_bib218","doi-asserted-by":"crossref","first-page":"41968","DOI":"10.1038\/srep41968","article-title":"A combinational CRISPR\/Cas9 gene-editing approach can halt HIV replication and prevent viral escape","volume":"7","author":"Lebbink","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib219","doi-asserted-by":"crossref","first-page":"1264","DOI":"10.1089\/hum.2018.018","article-title":"CRISPR\/Cas9 inhibits multiple steps of HIV-1 unfection","volume":"29","author":"Yin","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib220","doi-asserted-by":"crossref","first-page":"69","DOI":"10.3389\/fcimb.2019.00069","article-title":"Application of CRISPR\/Cas9-based gene editing in HIV-1\/AIDS therapy","volume":"9","author":"Xiao","year":"2019","journal-title":"Front. Cell. Infect. Microbiol."},{"key":"10.1016\/j.omtm.2020.06.022_bib221","doi-asserted-by":"crossref","first-page":"E114","DOI":"10.3390\/pharmaceutics11030114","article-title":"Gene therapy approaches to functional cure and protection of hematopoietic potential in HIV infection","volume":"11","author":"Tsukamoto","year":"2019","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.omtm.2020.06.022_bib222","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1186\/s13073-015-0174-y","article-title":"RNA interference approaches for treatment of HIV-1 infection","volume":"7","author":"Bobbin","year":"2015","journal-title":"Genome Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib223","doi-asserted-by":"crossref","first-page":"1301","DOI":"10.1084\/jem.20190287","article-title":"HIV-specific humoral immune responses by CRISPR\/Cas9-edited B cells","volume":"216","author":"Hartweger","year":"2019","journal-title":"J.\u00a0Exp. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib224","doi-asserted-by":"crossref","first-page":"e42995","DOI":"10.7554\/eLife.42995","article-title":"Reprogramming the antigen specificity of B cells using genome-editing technologies","volume":"8","author":"Voss","year":"2019","journal-title":"eLife"},{"key":"10.1016\/j.omtm.2020.06.022_bib225","doi-asserted-by":"crossref","first-page":"eaax0644","DOI":"10.1126\/sciimmunol.aax0644","article-title":"B cells engineered to express pathogen-specific antibodies protect against infection","volume":"4","author":"Moffett","year":"2019","journal-title":"Sci. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib226","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1038\/35066048","article-title":"Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias","volume":"2","author":"Weatherall","year":"2001","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.omtm.2020.06.022_bib227","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1182\/blood-2005-05-2113","article-title":"Regulation of human fetal hemoglobin: new players, new complexities","volume":"107","author":"Bank","year":"2006","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib228","doi-asserted-by":"crossref","first-page":"300","DOI":"10.1016\/j.blre.2018.02.001","article-title":"A paradigm shift on beta-thalassaemia treatment: how will we manage this old disease with new therapies?","volume":"32","author":"Cappellini","year":"2018","journal-title":"Blood Rev."},{"key":"10.1016\/j.omtm.2020.06.022_bib229","doi-asserted-by":"crossref","first-page":"100588","DOI":"10.1016\/j.blre.2019.100588","article-title":"Challenges of blood transfusions in \u03b2-thalassemia","volume":"37","author":"Shah","year":"2019","journal-title":"Blood Rev."},{"key":"10.1016\/j.omtm.2020.06.022_bib230","doi-asserted-by":"crossref","first-page":"1525","DOI":"10.1080\/03007995.2017.1326890","article-title":"Pattern of complications and burden of disease in patients affected by beta thalassemia major","volume":"33","author":"Bonifazi","year":"2017","journal-title":"Curr. Med. Res. Opin."},{"issue":"Suppl 1","key":"10.1016\/j.omtm.2020.06.022_bib231","doi-asserted-by":"crossref","first-page":"S76","DOI":"10.3109\/03630260903347336","article-title":"Update on survival in thalassemia major","volume":"33","author":"Telfer","year":"2009","journal-title":"Hemoglobin"},{"key":"10.1016\/j.omtm.2020.06.022_bib232","doi-asserted-by":"crossref","first-page":"515","DOI":"10.1097\/MOH.0000000000000280","article-title":"Transplantation for thalassemia major: alternative donors","volume":"23","author":"Locatelli","year":"2016","journal-title":"Curr. Opin. Hematol."},{"key":"10.1016\/j.omtm.2020.06.022_bib233","doi-asserted-by":"crossref","first-page":"178","DOI":"10.1159\/000358798","article-title":"Impact of HLA diversity on donor selection in organ and stem cell transplantation","volume":"76","author":"Tiercy","year":"2013","journal-title":"Hum. Hered."},{"key":"10.1016\/j.omtm.2020.06.022_bib234","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1517\/21678707.2015.1076724","article-title":"Cord blood transplantation in children with hemoglobinopathies","volume":"3","author":"Strocchio","year":"2015","journal-title":"Expert Opin. Orphan Drugs"},{"key":"10.1016\/j.omtm.2020.06.022_bib235","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1038\/bmt.2015.293","article-title":"Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000\u20132010","volume":"51","author":"Baronciani","year":"2016","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.omtm.2020.06.022_bib236","doi-asserted-by":"crossref","first-page":"1561","DOI":"10.1056\/NEJMra1510865","article-title":"Sickle cell disease","volume":"376","author":"Piel","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib237","doi-asserted-by":"crossref","first-page":"18010","DOI":"10.1038\/nrdp.2018.10","article-title":"Sickle cell disease","volume":"4","author":"Kato","year":"2018","journal-title":"Nat. Rev. Dis. Primers"},{"key":"10.1016\/j.omtm.2020.06.022_bib238","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1080\/17474086.2018.1486703","article-title":"Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced","volume":"11","author":"Leonard","year":"2018","journal-title":"Expert Rev. Hematol."},{"key":"10.1016\/j.omtm.2020.06.022_bib239","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1001\/jama.2014.7192","article-title":"Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype","volume":"312","author":"Hsieh","year":"2014","journal-title":"JAMA"},{"key":"10.1016\/j.omtm.2020.06.022_bib240","doi-asserted-by":"crossref","first-page":"2309","DOI":"10.1056\/NEJMoa0904971","article-title":"Allogeneic hematopoietic stem-cell transplantation for sickle cell disease","volume":"361","author":"Hsieh","year":"2009","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib241","doi-asserted-by":"crossref","first-page":"1171","DOI":"10.1016\/j.hoc.2014.08.014","article-title":"Hematopoietic stem cell transplantation for patients with sickle cell disease: progress and future directions","volume":"28","author":"Fitzhugh","year":"2014","journal-title":"Hematol. Oncol. Clin. North Am."},{"key":"10.1016\/j.omtm.2020.06.022_bib242","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1177\/2040620716653729","article-title":"The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges","volume":"7","author":"Goodman","year":"2016","journal-title":"Ther. Adv. Hematol."},{"key":"10.1016\/j.omtm.2020.06.022_bib243","doi-asserted-by":"crossref","first-page":"318","DOI":"10.1038\/nature09328","article-title":"Transfusion independence and HMGA2 activation after gene therapy of human \u03b2-thalassaemia","volume":"467","author":"Cavazzana-Calvo","year":"2010","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib244","doi-asserted-by":"crossref","first-page":"848","DOI":"10.1056\/NEJMoa1609677","article-title":"Gene therapy in a patient with sickle cell disease","volume":"376","author":"Ribeil","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib245","doi-asserted-by":"crossref","first-page":"1479","DOI":"10.1056\/NEJMoa1705342","article-title":"Gene therapy in patients with transfusion-dependent \u03b2-thalassemia","volume":"378","author":"Thompson","year":"2018","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib246","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1038\/s41591-018-0301-6","article-title":"Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent \u00df-thalassemia","volume":"25","author":"Marktel","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib247","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1126\/science.1091667","article-title":"Gene therapy insertional mutagenesis insights","volume":"303","author":"Dav\u00e9","year":"2004","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.06.022_bib248","doi-asserted-by":"crossref","first-page":"1550","DOI":"10.1001\/jama.2015.3253","article-title":"Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome","volume":"313","author":"Hacein-Bey Abina","year":"2015","journal-title":"JAMA"},{"key":"10.1016\/j.omtm.2020.06.022_bib249","doi-asserted-by":"crossref","first-page":"335ra57","DOI":"10.1126\/scitranslmed.aad8856","article-title":"Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency","volume":"8","author":"De Ravin","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib250","doi-asserted-by":"crossref","first-page":"1233158","DOI":"10.1126\/science.1233158","article-title":"Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy","volume":"341","author":"Biffi","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.06.022_bib251","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1016\/S0140-6736(16)30374-9","article-title":"Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1\/2 trial","volume":"388","author":"Sessa","year":"2016","journal-title":"Lancet"},{"key":"10.1016\/j.omtm.2020.06.022_bib252","doi-asserted-by":"crossref","first-page":"1630","DOI":"10.1056\/NEJMoa1700554","article-title":"Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy","volume":"377","author":"Eichler","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib253","doi-asserted-by":"crossref","first-page":"e239","DOI":"10.1016\/S2352-3026(19)30021-3","article-title":"Lentiviral haemopoietic stem\/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1\/2 clinical study","volume":"6","author":"Ferrua","year":"2019","journal-title":"Lancet Haematol."},{"key":"10.1016\/j.omtm.2020.06.022_bib254","doi-asserted-by":"crossref","first-page":"1525","DOI":"10.1056\/NEJMoa1815408","article-title":"Lentiviral gene therapy combined with low-dose busulfan in infants with SCID-X1","volume":"380","author":"Mamcarz","year":"2019","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib255","doi-asserted-by":"crossref","first-page":"917","DOI":"10.1016\/j.ymthe.2017.12.022","article-title":"Gene therapy for adenosine deaminase deficiency: a comprehensive evaluation of short- and medium-term safety","volume":"26","author":"Cicalese","year":"2018","journal-title":"Mol. Ther."},{"issue":"R2","key":"10.1016\/j.omtm.2020.06.022_bib256","doi-asserted-by":"crossref","first-page":"R216","DOI":"10.1093\/hmg\/ddp401","article-title":"Control of fetal hemoglobin: new insights emerging from genomics and clinical implications","volume":"18","author":"Thein","year":"2009","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2020.06.022_bib257","doi-asserted-by":"crossref","first-page":"430","DOI":"10.1016\/j.cell.2018.03.016","article-title":"Direct promoter repression by BCL11A controls the fetal to adult hemoglobin switch","volume":"173","author":"Liu","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2020.06.022_bib258","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1016\/j.bcmd.2008.12.003","article-title":"BCL11A represses HBG transcription in K562 cells","volume":"42","author":"Chen","year":"2009","journal-title":"Blood Cells Mol. Dis."},{"key":"10.1016\/j.omtm.2020.06.022_bib259","doi-asserted-by":"crossref","first-page":"1093","DOI":"10.1038\/nature08243","article-title":"Developmental and species-divergent globin switching are driven by BCL11A","volume":"460","author":"Sankaran","year":"2009","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib260","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1016\/j.omtm.2016.12.009","article-title":"Long-term engraftment and fetal globin induction upon BCL11A gene editing in bone-marrow-derived CD34+ hematopoietic stem and progenitor cells","volume":"4","author":"Chang","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2020.06.022_bib261","doi-asserted-by":"crossref","first-page":"2338","DOI":"10.1182\/blood-2016-08-736249","article-title":"Strict in\u00a0vivo specificity of the Bcl11a erythroid enhancer","volume":"128","author":"Smith","year":"2016","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib262","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1016\/j.omtm.2018.08.003","article-title":"Disruption of the BCL11A erythroid enhancer reactivates fetal hemoglobin in erythroid cells of patients with \u03b2-thalassemia major","volume":"10","author":"Psatha","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2020.06.022_bib263","unstructured":"Chapin, J.; Vertex Pharmaceuticals Incorporated; CRISPR Therapeutics (2018) A safety and efficacy study evaluating CTX001 in subjects with transfusion-dependent \u03b2-thalassemia. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03655678."},{"key":"10.1016\/j.omtm.2020.06.022_bib264","unstructured":"Schiller, G., Walters, M., Williams, D., and Smith, A; Sangamo Therapeutics; Sanofi. (2018). A study to assess the safety, tolerability, and efficacy of ST-400 for treatment of transfusion-dependent beta-thalassemia (TDT). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03432364."},{"key":"10.1016\/j.omtm.2020.06.022_bib265","unstructured":"Chapin, J.; Vertex Pharmaceuticals Incorporated; CRISPR Therapeutics (2018). A safety and efficacy study evaluating CTX001 in subjects with severe sickle cell disease. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03745287."},{"key":"10.1016\/j.omtm.2020.06.022_bib266","unstructured":"Sanofi; Bioverativ Therapeutics Inc. (2018). A study to assess the safety, tolerability, and efficacy of BIVV003 for autologous hematopoietic stem cell transplantation in patients with severe sickle cell disease (BIVV003). https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03653247."},{"key":"10.1016\/j.omtm.2020.06.022_bib267","unstructured":"Vertex Pharmaceuticals Incorporated; CRISPR Therapeutics (2019). A long-term follow-up study in subjects who received CTX001. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04208529."},{"key":"10.1016\/j.omtm.2020.06.022_bib268","doi-asserted-by":"crossref","first-page":"987","DOI":"10.1038\/nm.4170","article-title":"A genome-editing strategy to treat \u03b2-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition","volume":"22","author":"Traxler","year":"2016","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib269","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1016\/j.omtm.2018.12.008","article-title":"TALEN-mediated gene editing of HBG in human hematopoietic stem cells leads to therapeutic fetal hemoglobin induction","volume":"12","author":"Lux","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2020.06.022_bib270","doi-asserted-by":"crossref","first-page":"10661","DOI":"10.1073\/pnas.1612075113","article-title":"Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: an approach for treating sickle cell disease and \u03b2-thalassemia","volume":"113","author":"Ye","year":"2016","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2020.06.022_bib271","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1002\/sctm.17-0066","article-title":"A universal approach to correct various HBB gene mutations in human stem cells for gene therapy of beta-thalassemia and sickle cell disease","volume":"7","author":"Cai","year":"2018","journal-title":"Stem Cells Transl. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib272","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1186\/s13287-018-0779-3","article-title":"One-step genetic correction of hemoglobin E\/beta-thalassemia patient-derived iPSCs by the CRISPR\/Cas9 system","volume":"9","author":"Wattanapanitch","year":"2018","journal-title":"Stem Cell Res. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib273","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1016\/j.stem.2019.04.001","article-title":"Highly efficient and marker-free genome editing of human pluripotent stem cells by CRISPR-Cas9 RNP and AAV6 donor-mediated homologous recombination","volume":"24","author":"Martin","year":"2019","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.omtm.2020.06.022_bib274","doi-asserted-by":"crossref","first-page":"7955","DOI":"10.1093\/nar\/gkz475","article-title":"Highly efficient editing of the \u03b2-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease","volume":"47","author":"Park","year":"2019","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib275","doi-asserted-by":"crossref","first-page":"1526","DOI":"10.1101\/gr.173427.114","article-title":"Seamless gene correction of \u03b2-thalassemia mutations in patient-specific iPSCs using CRISPR\/Cas9 and piggyBac","volume":"24","author":"Xie","year":"2014","journal-title":"Genome Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib276","doi-asserted-by":"crossref","first-page":"1053","DOI":"10.1089\/scd.2014.0347","article-title":"Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR\/Cas9 system","volume":"24","author":"Song","year":"2015","journal-title":"Stem Cells Dev."},{"key":"10.1016\/j.omtm.2020.06.022_bib277","doi-asserted-by":"crossref","first-page":"12065","DOI":"10.1038\/srep12065","article-title":"Both TALENs and CRISPR\/Cas9 directly target the HBB IVS2-654 (C > T) mutation in \u03b2-thalassemia-derived iPSCs","volume":"5","author":"Xu","year":"2015","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib278","doi-asserted-by":"crossref","first-page":"16576","DOI":"10.1074\/jbc.M116.719237","article-title":"Combining single strand oligodeoxynucleotides and CRISPR\/Cas9 to correct gene mutations in \u03b2-thalassemia-induced pluripotent stem cells","volume":"291","author":"Niu","year":"2016","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2020.06.022_bib279","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1016\/j.omtn.2016.11.010","article-title":"One-step biallelic and scarless correction of a \u03b2-thalassemia mutation in patient-specific iPSCs without Drug Selection","volume":"6","author":"Liu","year":"2017","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2020.06.022_bib280","unstructured":"Allife Medical Science and Technology Co., Ltd. (2018). iHSCs with the gene correction of HBB intervent subjests with \u03b2-thalassemia mutations. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03728322."},{"key":"10.1016\/j.omtm.2020.06.022_bib281","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1038\/s41467-017-00479-7","article-title":"Editing an \u03b1-globin enhancer in primary human hematopoietic stem cells as a treatment for \u03b2-thalassemia","volume":"8","author":"Mettananda","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2020.06.022_bib282","doi-asserted-by":"crossref","first-page":"1013","DOI":"10.1089\/hum.2017.116","article-title":"Hemophilia gene therapy: ready for prime time?","volume":"28","author":"VandenDriessche","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib283","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1186\/1750-1172-7-24","article-title":"Past, present and future of hemophilia: a narrative review","volume":"7","author":"Franchini","year":"2012","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2020.06.022_bib284","doi-asserted-by":"crossref","first-page":"2038","DOI":"10.1182\/blood-2003-01-0167","article-title":"Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion","volume":"102","author":"Powell","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib285","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1038\/73464","article-title":"Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector","volume":"24","author":"Kay","year":"2000","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtm.2020.06.022_bib286","doi-asserted-by":"crossref","first-page":"342","DOI":"10.1038\/nm1358","article-title":"Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response","volume":"12","author":"Manno","year":"2006","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib287","doi-asserted-by":"crossref","first-page":"1735","DOI":"10.1056\/NEJM200106073442301","article-title":"Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A","volume":"344","author":"Roth","year":"2001","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib288","doi-asserted-by":"crossref","first-page":"1777","DOI":"10.1182\/blood-2014-12-615492","article-title":"In\u00a0vivo genome editing of the albumin locus as a platform for protein replacement therapy","volume":"126","author":"Sharma","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib289","unstructured":"Quon, D., Kuriakose, P.; Sangamo Therapeutics (2016). Ascending dose study of genome editing by zinc finger nuclease therapeutic SB-FIX in subjects with severe hemophilia B. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02695160."},{"key":"10.1016\/j.omtm.2020.06.022_bib290","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1186\/s13287-018-0839-8","article-title":"Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system","volume":"9","author":"Lyu","year":"2018","journal-title":"Stem Cell Res. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib291","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1038\/mt.2015.223","article-title":"Multidimensional genome-wide analyses show accurate FVIII integration by ZFN in primary human cells","volume":"24","author":"Sivalingam","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib292","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1038\/nature10177","article-title":"In\u00a0vivo genome editing restores haemostasis in a mouse model of haemophilia","volume":"475","author":"Li","year":"2011","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib293","doi-asserted-by":"crossref","first-page":"3283","DOI":"10.1182\/blood-2013-04-497354","article-title":"Robust ZFN-mediated genome editing in adult hemophilic mice","volume":"122","author":"Anguela","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2020.06.022_bib294","doi-asserted-by":"crossref","first-page":"e3020","DOI":"10.1002\/jgm.3020","article-title":"Designer nuclease-mediated gene correction via homology-directed repair in an in\u00a0vitro model of canine hemophilia B","volume":"20","author":"Bergmann","year":"2018","journal-title":"J.\u00a0Gene Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib295","doi-asserted-by":"crossref","first-page":"4159","DOI":"10.1038\/s41598-017-04625-5","article-title":"CRISPR\/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice","volume":"7","author":"Ohmori","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib296","doi-asserted-by":"crossref","first-page":"477","DOI":"10.15252\/emmm.201506039","article-title":"CRISPR\/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse","volume":"8","author":"Guan","year":"2016","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib297","doi-asserted-by":"crossref","first-page":"135","DOI":"10.24920\/J1001-9294.2017.032","article-title":"Genetic correction and hepatic differentiation of hemophilia B-specific human induced pluripotent stem cells","volume":"32","author":"He","year":"2017","journal-title":"Chin. Med. Sci. J."},{"key":"10.1016\/j.omtm.2020.06.022_bib298","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1007\/s00439-017-1801-z","article-title":"CRISPR\/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice","volume":"136","author":"Huai","year":"2017","journal-title":"Hum. Genet."},{"key":"10.1016\/j.omtm.2020.06.022_bib299","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.stem.2015.07.001","article-title":"Functional correction of large factor VIII gene chromosomal inversions in hemophilia A patient-derived iPSCs using CRISPR-Cas9","volume":"17","author":"Park","year":"2015","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.omtm.2020.06.022_bib300","doi-asserted-by":"crossref","first-page":"9253","DOI":"10.1073\/pnas.1323941111","article-title":"Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs","volume":"111","author":"Park","year":"2014","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2020.06.022_bib301","doi-asserted-by":"crossref","first-page":"2154","DOI":"10.1038\/nprot.2016.129","article-title":"Modeling and correction of structural variations in patient-derived iPSCs using CRISPR\/Cas9","volume":"11","author":"Park","year":"2016","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.omtm.2020.06.022_bib302","doi-asserted-by":"crossref","first-page":"4730","DOI":"10.1038\/s41467-019-12726-0","article-title":"Functional profiling of single CRISPR\/Cas9-edited human long-term hematopoietic stem cells","volume":"10","author":"Wagenblast","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2020.06.022_bib303","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1038\/nature13420","article-title":"Targeted genome editing in human repopulating haematopoietic stem cells","volume":"510","author":"Genovese","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2020.06.022_bib304","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1038\/nbt.3513","article-title":"Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease","volume":"34","author":"De Ravin","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.06.022_bib305","doi-asserted-by":"crossref","first-page":"eaan0820","DOI":"10.1126\/scitranslmed.aan0820","article-title":"Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1","volume":"9","author":"Schiroli","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib306","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1016\/j.ymgme.2017.12.434","article-title":"Gene therapy for mucopolysaccharidoses","volume":"123","author":"Sawamoto","year":"2018","journal-title":"Mol. Genet. Metab."},{"issue":"Suppl 2","key":"10.1016\/j.omtm.2020.06.022_bib307","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1186\/s13052-018-0562-1","article-title":"Enzyme replacement therapy: efficacy and limitations","volume":"44","author":"Concolino","year":"2018","journal-title":"Ital. J. Pediatr."},{"key":"10.1016\/j.omtm.2020.06.022_bib308","doi-asserted-by":"crossref","first-page":"94","DOI":"10.1016\/j.ymgme.2014.12.001","article-title":"Therapies for the bone in mucopolysaccharidoses","volume":"114","author":"Tomatsu","year":"2015","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2020.06.022_bib309","doi-asserted-by":"crossref","first-page":"e226","DOI":"10.1016\/j.bbmt.2019.02.012","article-title":"Hematopoietic stem cell transplantation for mucopolysaccharidoses: past, present, and future","volume":"25","author":"Taylor","year":"2019","journal-title":"Biol. Blood Marrow Transplant."},{"key":"10.1016\/j.omtm.2020.06.022_bib310","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1016\/j.jconrel.2018.08.031","article-title":"In\u00a0vivo genome editing of mucopolysaccharidosis I mice using the CRISPR\/Cas9 system","volume":"288","author":"Schuh","year":"2018","journal-title":"J.\u00a0Control. Release"},{"key":"10.1016\/j.omtm.2020.06.022_bib311","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1038\/mt.2010.265","article-title":"Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes","volume":"19","author":"Ellinwood","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib312","doi-asserted-by":"crossref","first-page":"e86696","DOI":"10.1172\/jci.insight.86696","article-title":"CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)","volume":"1","author":"Motas","year":"2016","journal-title":"JCI Insight"},{"key":"10.1016\/j.omtm.2020.06.022_bib313","doi-asserted-by":"crossref","first-page":"1025","DOI":"10.1038\/mt.2011.34","article-title":"Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery","volume":"19","author":"Fu","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib314","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1002\/emmm.201202083","article-title":"A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA","volume":"5","author":"Sorrentino","year":"2013","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib315","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1038\/sj.mt.6300325","article-title":"Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle","volume":"16","author":"Tessitore","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib316","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1038\/mt.2015.189","article-title":"Evaluation of AAV-mediated gene therapy for central nervous system disease in canine mucopolysaccharidosis VII","volume":"24","author":"Gurda","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib317","doi-asserted-by":"crossref","first-page":"712","DOI":"10.1016\/S1474-4422(17)30169-2","article-title":"Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1\/2 clinical trial","volume":"16","author":"Tardieu","year":"2017","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.omtm.2020.06.022_bib318","doi-asserted-by":"crossref","first-page":"1127","DOI":"10.1016\/j.ymthe.2018.03.002","article-title":"Dose-dependent prevention of metabolic and neurologic disease in murine MPS II by ZFN-mediated in\u00a0vivo genome editing","volume":"26","author":"Laoharawee","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib319","unstructured":"Harmatz, P., Heldermon, C., Wilcox, W., Whitley, C., Lau, H., and Leslie, N.; Sangamo Therapeutics (2018). Ascending dose study of genome editing by the zinc finger nuclease (ZFN) therapeutic SB-318 in subjects with MPS I. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02702115."},{"key":"10.1016\/j.omtm.2020.06.022_bib320","unstructured":"Burton, B., Whitley, C., Lau, H., Muenzer, J., Prada, C., and Ficcioglu, C; Sangamo Therapeutics (2017). Ascending dose study of genome editing by the zinc finger nuclease (ZFN) therapeutic SB-913 in subjects with MPS II. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03041324."},{"key":"10.1016\/j.omtm.2020.06.022_bib321","doi-asserted-by":"crossref","first-page":"1147","DOI":"10.1136\/bjophthalmol-2016-309975","article-title":"Leber congenital amaurosis\/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions","volume":"101","author":"Kumaran","year":"2017","journal-title":"Br. J. Ophthalmol."},{"key":"10.1016\/j.omtm.2020.06.022_bib322","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1016\/j.preteyeres.2017.05.001","article-title":"Cilia\u2014the sensory antennae in the eye","volume":"60","author":"May-Simera","year":"2017","journal-title":"Prog. Retin. Eye Res."},{"key":"10.1016\/j.omtm.2020.06.022_bib323","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1001\/archophthalmol.2011.298","article-title":"Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years","volume":"130","author":"Jacobson","year":"2012","journal-title":"Arch. Ophthalmol."},{"key":"10.1016\/j.omtm.2020.06.022_bib324","doi-asserted-by":"crossref","first-page":"2231","DOI":"10.1056\/NEJMoa0802268","article-title":"Effect of gene therapy on visual function in Leber\u2019s congenital amaurosis","volume":"358","author":"Bainbridge","year":"2008","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib325","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","article-title":"Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial","volume":"390","author":"Russell","year":"2017","journal-title":"Lancet"},{"key":"10.1016\/j.omtm.2020.06.022_bib326","doi-asserted-by":"crossref","first-page":"2240","DOI":"10.1056\/NEJMoa0802315","article-title":"Safety and efficacy of gene transfer for Leber\u2019s congenital amaurosis","volume":"358","author":"Maguire","year":"2008","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib327","first-page":"480","article-title":"Leber\u2019s congenital amaurosis and the role of gene therapy in congenital retinal disorders","volume":"10","author":"Sharif","year":"2017","journal-title":"Int. J. Ophthalmol."},{"key":"10.1016\/j.omtm.2020.06.022_bib328","doi-asserted-by":"crossref","first-page":"46","DOI":"10.3389\/fcell.2018.00046","article-title":"Translation of CRISPR genome surgery to the bedside for retinal diseases","volume":"6","author":"Xu","year":"2018","journal-title":"Front. Cell Dev. Biol."},{"key":"10.1016\/j.omtm.2020.06.022_bib329","unstructured":"Allergan; Editas Medicine, Inc. (2019). Single ascending dose study in participants with LCA10. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03872479."},{"key":"10.1016\/j.omtm.2020.06.022_bib330","doi-asserted-by":"crossref","first-page":"1097","DOI":"10.1002\/humu.21337","article-title":"CEP290, a gene with many faces: mutation overview and presentation of CEP290base","volume":"31","author":"Coppieters","year":"2010","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.omtm.2020.06.022_bib331","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/j.ymthe.2016.12.006","article-title":"CRISPR\/Cas9-mediated genome editing as a therapeutic approach for Leber congenital amaurosis 10","volume":"25","author":"Ruan","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.06.022_bib332","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1038\/s41591-018-0327-9","article-title":"Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10","volume":"25","author":"Maeder","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib333","doi-asserted-by":"crossref","first-page":"5056279","DOI":"10.1155\/2018\/5056279","article-title":"Guiding lights in genome editing for inherited retinal disorders: implications for gene and cell therapy","volume":"2018","author":"Sanjurjo-Soriano","year":"2018","journal-title":"Neural Plast."},{"key":"10.1016\/j.omtm.2020.06.022_bib334","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1016\/j.ddtec.2018.05.001","article-title":"CRISPR\/Cas9 genome surgery for retinal diseases","volume":"28","author":"Xu","year":"2018","journal-title":"Drug Discov. Today. Technol."},{"key":"10.1016\/j.omtm.2020.06.022_bib335","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1016\/j.gendis.2019.07.007","article-title":"Gene therapy and genome editing for primary immunodeficiency diseases","volume":"7","author":"Zhang","year":"2019","journal-title":"Genes Dis."},{"issue":"R1","key":"10.1016\/j.omtm.2020.06.022_bib336","doi-asserted-by":"crossref","first-page":"R15","DOI":"10.1093\/hmg\/ddz170","article-title":"Gene therapy for primary immunodeficiency","volume":"28","author":"Booth","year":"2019","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2020.06.022_bib337","doi-asserted-by":"crossref","first-page":"558","DOI":"10.1002\/hep4.1315","article-title":"Ex\u00a0vivo hepatocyte reprograming promotes homology-directed DNA repair to correct metabolic disease in mice after transplantation","volume":"3","author":"VanLith","year":"2019","journal-title":"Hepatol. Commun."},{"key":"10.1016\/j.omtm.2020.06.022_bib338","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1016\/j.stem.2019.11.002","article-title":"High-efficiency, selection-free gene repair in airway stem cells from cystic fibrosis patients rescues CFTR function in differentiated epithelia","volume":"26","author":"Vaidyanathan","year":"2020","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.omtm.2020.06.022_bib339","doi-asserted-by":"crossref","first-page":"e49612","DOI":"10.15252\/embr.201949612","article-title":"Mitochondrial DNA heteroplasmy in disease and targeted nuclease-based therapeutic approaches","volume":"21","author":"Nissanka","year":"2020","journal-title":"EMBO Rep."},{"key":"10.1016\/j.omtm.2020.06.022_bib340","doi-asserted-by":"crossref","first-page":"685","DOI":"10.1111\/joim.13055","article-title":"DNA-editing enzymes as potential treatments for heteroplasmic mtDNA diseases","volume":"287","author":"Zekonyte","year":"2020","journal-title":"J.\u00a0Intern. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib341","doi-asserted-by":"crossref","first-page":"12642","DOI":"10.1038\/ncomms12642","article-title":"Reprogramming metabolic pathways in\u00a0vivo with CRISPR\/Cas9 genome editing to treat hereditary tyrosinaemia","volume":"7","author":"Pankowicz","year":"2016","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2020.06.022_bib342","doi-asserted-by":"crossref","first-page":"1519","DOI":"10.1038\/s41591-018-0209-1","article-title":"Treatment of a metabolic liver disease by in\u00a0vivo genome base editing in adult mice","volume":"24","author":"Villiger","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.06.022_bib343","doi-asserted-by":"crossref","first-page":"3060","DOI":"10.3389\/fimmu.2019.03060","article-title":"Xenotransplantation: current status in preclinical research","volume":"10","author":"Lu","year":"2020","journal-title":"Front. Immunol."},{"key":"10.1016\/j.omtm.2020.06.022_bib344","doi-asserted-by":"crossref","DOI":"10.1038\/s41587-020-0551-y","article-title":"Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking","author":"Ferrari","year":"2020","journal-title":"Nat. Biotechnol."}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050120301467?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050120301467?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:29:49Z","timestamp":1762421389000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050120301467"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,9]]},"references-count":344,"alternative-id":["S2329050120301467"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2020.06.022","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2020,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2020.06.022","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2020 The Authors.","name":"copyright","label":"Copyright"}]}}