{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T03:11:50Z","timestamp":1792120310872,"version":"4.4.0"},"reference-count":56,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2021,6,1]],"date-time":"2021-06-01T00:00:00Z","timestamp":1622505600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2021,6,1]],"date-time":"2021-06-01T00:00:00Z","timestamp":1622505600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,2,26]],"date-time":"2021-02-26T00:00:00Z","timestamp":1614297600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000062","name":"National Institute of Diabetes and Digestive and Kidney Diseases","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000062","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["WO 2017\/059241"],"award-info":[{"award-number":["WO 2017\/059241"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2021,6]]},"DOI":"10.1016\/j.omtm.2021.02.022","type":"journal-article","created":{"date-parts":[[2021,3,2]],"date-time":"2021-03-02T22:38:24Z","timestamp":1614724704000},"page":"121-132","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":53,"special_numbering":"C","title":["Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease"],"prefix":"10.1016","volume":"21","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6322-8991","authenticated-orcid":false,"given":"Naoya","family":"Uchida","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claire M.","family":"Drysdale","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8576-9737","authenticated-orcid":false,"given":"Tina","family":"Nassehi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6547-3568","authenticated-orcid":false,"given":"Jackson","family":"Gamer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1016-2153","authenticated-orcid":false,"given":"Morgan","family":"Yapundich","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7076-8259","authenticated-orcid":false,"given":"Julia","family":"DiNicola","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0107-2877","authenticated-orcid":false,"given":"Yoshitaka","family":"Shibata","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Malikiya","family":"Hinds","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1236-3410","authenticated-orcid":false,"given":"Bjorg","family":"Gudmundsdottir","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan J.","family":"Haro-Mora","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Selami","family":"Demirci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John F.","family":"Tisdale","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2021.02.022_bib1","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1056\/NEJMoa0805817","article-title":"Gene therapy for immunodeficiency due to adenosine deaminase deficiency","volume":"360","author":"Aiuti","year":"2009","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.02.022_bib2","doi-asserted-by":"crossref","first-page":"2410","DOI":"10.1126\/science.1070104","article-title":"Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning","volume":"296","author":"Aiuti","year":"2002","journal-title":"Science"},{"key":"10.1016\/j.omtm.2021.02.022_bib3","doi-asserted-by":"crossref","first-page":"1918","DOI":"10.1056\/NEJMoa1003548","article-title":"Stem-cell gene therapy for the Wiskott-Aldrich syndrome","volume":"363","author":"Boztug","year":"2010","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.02.022_bib4","doi-asserted-by":"crossref","first-page":"669","DOI":"10.1126\/science.288.5466.669","article-title":"Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease","volume":"288","author":"Cavazzana-Calvo","year":"2000","journal-title":"Science"},{"key":"10.1016\/j.omtm.2021.02.022_bib5","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.2021.02.022_bib6","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1056\/NEJM200301163480314","article-title":"A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency","volume":"348","author":"Hacein-Bey-Abina","year":"2003","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.02.022_bib7","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.2021.02.022_bib8","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1126\/science.1179555","article-title":"CRISPR\/Cas, the immune system of bacteria and archaea","volume":"327","author":"Horvath","year":"2010","journal-title":"Science"},{"key":"10.1016\/j.omtm.2021.02.022_bib9","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1038\/nbt.2501","article-title":"Efficient genome editing in zebrafish using a CRISPR-Cas system","volume":"31","author":"Hwang","year":"2013","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2021.02.022_bib10","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.2021.02.022_bib11","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1038\/nprot.2017.143","article-title":"CRISPR\/Cas9 genome editing in human hematopoietic stem cells","volume":"13","author":"Bak","year":"2018","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.omtm.2021.02.022_bib12","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1016\/j.ymthe.2018.10.008","article-title":"Optimization of CRISPR\/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements","volume":"27","author":"Lattanzi","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib13","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1038\/nature20134","article-title":"CRISPR\/Cas9 \u03b2-globin gene targeting in human haematopoietic stem cells","volume":"539","author":"Dever","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2021.02.022_bib14","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1038\/nrg3686","article-title":"A guide to genome engineering with programmable nucleases","volume":"15","author":"Kim","year":"2014","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.omtm.2021.02.022_bib15","doi-asserted-by":"crossref","first-page":"360ra134","DOI":"10.1126\/scitranslmed.aaf9336","article-title":"Selection-free genome editing of the sickle mutation in human adult hematopoietic stem\/progenitor cells","volume":"8","author":"DeWitt","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2021.02.022_bib16","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1016\/j.ymthe.2019.05.014","article-title":"Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates","volume":"27","author":"Romero","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib17","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1016\/j.omtn.2019.05.025","article-title":"In\u00a0Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods","volume":"17","author":"Pattabhi","year":"2019","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2021.02.022_bib18","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.2021.02.022_bib19","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1111\/bjh.15902","article-title":"Bone marrow characterization in sickle cell disease: inflammation and stress erythropoiesis lead to suboptimal CD34 recovery","volume":"186","author":"Leonard","year":"2019","journal-title":"Br. J. Haematol."},{"key":"10.1016\/j.omtm.2021.02.022_bib20","doi-asserted-by":"crossref","first-page":"1882","DOI":"10.1038\/mt.2012.159","article-title":"High-efficiency transduction of rhesus hematopoietic repopulating cells by a modified HIV1-based lentiviral vector","volume":"20","author":"Uchida","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib21","doi-asserted-by":"crossref","first-page":"1078","DOI":"10.1038\/gt.2011.63","article-title":"Optimal conditions for lentiviral transduction of engrafting human CD34+ cells","volume":"18","author":"Uchida","year":"2011","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib22","doi-asserted-by":"crossref","first-page":"4479","DOI":"10.1038\/s41467-019-12456-3","article-title":"Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders","volume":"10","author":"Uchida","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2021.02.022_bib23","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1016\/j.omtm.2019.01.005","article-title":"High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2","volume":"13","author":"Uchida","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.02.022_bib24","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1038\/nmeth.3047","article-title":"Improved vectors and genome-wide libraries for CRISPR screening","volume":"11","author":"Sanjana","year":"2014","journal-title":"Nat. Methods"},{"key":"10.1016\/j.omtm.2021.02.022_bib25","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1016\/j.omtm.2017.04.002","article-title":"Integrase-Deficient Lentiviral Vector as an All-in-One Platform for Highly Efficient CRISPR\/Cas9-Mediated Gene Editing","volume":"5","author":"Ortinski","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.02.022_bib26","doi-asserted-by":"crossref","first-page":"3132","DOI":"10.1172\/JCI35700","article-title":"Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1","volume":"118","author":"Hacein-Bey-Abina","year":"2008","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2021.02.022_bib27","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1126\/science.1088547","article-title":"LMO2-associated clonal T\u00a0cell proliferation in two patients after gene therapy for SCID-X1","volume":"302","author":"Hacein-Bey-Abina","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.omtm.2021.02.022_bib28","doi-asserted-by":"crossref","first-page":"9854","DOI":"10.1128\/JVI.00357-09","article-title":"Development of a human immunodeficiency virus type 1-based lentiviral vector that allows efficient transduction of both human and rhesus blood cells","volume":"83","author":"Uchida","year":"2009","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2021.02.022_bib29","doi-asserted-by":"crossref","first-page":"4299","DOI":"10.1128\/jvi.70.7.4299-4310.1996","article-title":"Binding of the human immunodeficiency virus type 1 Gag polyprotein to cyclophilin A is mediated by the central region of capsid and requires Gag dimerization","volume":"70","author":"Colgan","year":"1996","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2021.02.022_bib30","doi-asserted-by":"crossref","first-page":"468","DOI":"10.1016\/j.ymthe.2017.11.001","article-title":"Characterization of Gene Alterations following Editing of the \u03b2-Globin Gene Locus in Hematopoietic Stem\/Progenitor Cells","volume":"26","author":"Long","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib31","doi-asserted-by":"crossref","first-page":"1431","DOI":"10.1038\/sj.gt.3301252","article-title":"High-level transgene expression in primary human T lymphocytes and adult bone marrow CD34+ cells via electroporation-mediated gene delivery","volume":"7","author":"Van Tendeloo","year":"2000","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib32","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1038\/sj.gt.3301393","article-title":"Efficient expression of foreign genes in human CD34(+) hematopoietic precursor cells using electroporation","volume":"8","author":"Wu","year":"2001","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib33","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1634\/stemcells.19-6-492","article-title":"High efficiency electroporation of human umbilical cord blood CD34+ hematopoietic precursor cells","volume":"19","author":"Wu","year":"2001","journal-title":"Stem Cells"},{"key":"10.1016\/j.omtm.2021.02.022_bib34","doi-asserted-by":"crossref","first-page":"1012","DOI":"10.1101\/gr.171322.113","article-title":"Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins","volume":"24","author":"Kim","year":"2014","journal-title":"Genome Res."},{"key":"10.1016\/j.omtm.2021.02.022_bib35","doi-asserted-by":"crossref","first-page":"e122","DOI":"10.1038\/mtna.2013.49","article-title":"Integration-specific In\u00a0Vitro Evaluation of Lentivirally Transduced Rhesus CD34(+) Cells Correlates With In\u00a0Vivo Vector Copy Number","volume":"2","author":"Uchida","year":"2013","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2021.02.022_bib36","doi-asserted-by":"crossref","first-page":"721","DOI":"10.1128\/jvi.70.2.721-728.1996","article-title":"Human immunodeficiency virus type 1 integrase mutants retain in\u00a0vitro integrase activity yet fail to integrate viral DNA efficiently during infection","volume":"70","author":"Leavitt","year":"1996","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2021.02.022_bib37","doi-asserted-by":"crossref","first-page":"1316","DOI":"10.1038\/mt.2009.122","article-title":"Integration-deficient lentiviral vectors: a slow coming of age","volume":"17","author":"Wanisch","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib38","doi-asserted-by":"crossref","first-page":"912","DOI":"10.1016\/j.omtm.2020.04.013","article-title":"\u03b2T87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex\u00a0Vivo Anti-sickling Activity in Human Erythroid Cells","volume":"17","author":"Demirci","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"issue":"Suppl 1","key":"10.1016\/j.omtm.2021.02.022_bib39","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1182\/blood-2019-122727","article-title":"Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model","volume":"134","author":"Uchida","year":"2019","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.02.022_bib40","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1038\/nbt.4021","article-title":"Inhibition of 53BP1 favors homology-dependent DNA repair and increases CRISPR-Cas9 genome-editing efficiency","volume":"36","author":"Canny","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2021.02.022_bib41","doi-asserted-by":"crossref","first-page":"2866","DOI":"10.1038\/s41467-019-10735-7","article-title":"CRISPR-Cas9 fusion to dominant-negative 53BP1 enhances HDR and inhibits NHEJ specifically at Cas9 target sites","volume":"10","author":"Jayavaradhan","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2021.02.022_bib42","doi-asserted-by":"crossref","first-page":"453","DOI":"10.1016\/j.omtn.2018.05.010","article-title":"VSV-G-Enveloped Vesicles for Traceless Delivery of CRISPR-Cas9","volume":"12","author":"Montagna","year":"2018","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2021.02.022_bib43","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1038\/s41467-018-07845-z","article-title":"Genome editing in primary cells and in\u00a0vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins","volume":"10","author":"Mangeot","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2021.02.022_bib44","doi-asserted-by":"crossref","first-page":"1298","DOI":"10.1038\/nbt1353","article-title":"Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery","volume":"25","author":"Lombardo","year":"2007","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2021.02.022_bib45","doi-asserted-by":"crossref","first-page":"2597","DOI":"10.1182\/blood-2014-12-615948","article-title":"Correction of the sickle cell disease mutation in human hematopoietic stem\/progenitor cells","volume":"125","author":"Hoban","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.02.022_bib46","doi-asserted-by":"crossref","first-page":"1334","DOI":"10.1038\/s41467-020-14957-y","article-title":"Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping","volume":"11","author":"Gee","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2021.02.022_bib47","doi-asserted-by":"crossref","first-page":"242","DOI":"10.1006\/mthe.2002.0549","article-title":"Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood","volume":"5","author":"Hanawa","year":"2002","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib48","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1272\/jnms.76.134","article-title":"Leukemogenesis of b2a2-type p210 BCR\/ABL in a bone marrow transplantation mouse model using a lentiviral vector","volume":"76","author":"Uchida","year":"2009","journal-title":"J.\u00a0Nippon Med. Sch."},{"key":"10.1016\/j.omtm.2021.02.022_bib49","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1126\/science.1231143","article-title":"Multiplex genome engineering using CRISPR\/Cas systems","volume":"339","author":"Cong","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.omtm.2021.02.022_bib50","doi-asserted-by":"crossref","first-page":"822","DOI":"10.1038\/nbt.2623","article-title":"High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells","volume":"31","author":"Fu","year":"2013","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2021.02.022_bib51","doi-asserted-by":"crossref","first-page":"e59890","DOI":"10.1371\/journal.pone.0059890","article-title":"Establishment of immortalized human erythroid progenitor cell lines able to produce enucleated red blood cells","volume":"8","author":"Kurita","year":"2013","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2021.02.022_bib52","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1016\/j.omtm.2018.03.007","article-title":"Serum-free Erythroid Differentiation for Efficient Genetic Modification and High-Level Adult Hemoglobin Production","volume":"9","author":"Uchida","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.02.022_bib53","doi-asserted-by":"crossref","first-page":"586","DOI":"10.1002\/stem.2517","article-title":"Efficient Generation of \u03b2-Globin-Expressing Erythroid Cells Using Stromal Cell-Derived Induced Pluripotent Stem Cells from Patients with Sickle Cell Disease","volume":"35","author":"Uchida","year":"2017","journal-title":"Stem Cells"},{"key":"10.1016\/j.omtm.2021.02.022_bib54","doi-asserted-by":"crossref","first-page":"224","DOI":"10.1038\/s41587-019-0032-3","article-title":"CRISPResso2 provides accurate and rapid genome editing sequence analysis","volume":"37","author":"Clement","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2021.02.022_bib55","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1038\/mt.2013.256","article-title":"TRIM5alpha variations influence transduction efficiency with lentiviral vectors in both human and rhesus CD34(+) cells in\u00a0vitro and in\u00a0vivo","volume":"22","author":"Evans","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.02.022_bib56","doi-asserted-by":"crossref","first-page":"2667","DOI":"10.1128\/JVI.73.4.2667-2674.1999","article-title":"Vif and the p55(Gag) polyprotein of human immunodeficiency virus type 1 are present in colocalizing membrane-free cytoplasmic complexes","volume":"73","author":"Simon","year":"1999","journal-title":"J.\u00a0Virol."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121000371?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121000371?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,15]],"date-time":"2026-05-15T17:52:27Z","timestamp":1778867547000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050121000371"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,6]]},"references-count":56,"alternative-id":["S2329050121000371"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2021.02.022","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2021,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell 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.2021.02.022","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"name":"copyright","label":"Copyright"}]}}