{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T03:17:01Z","timestamp":1787887021512,"version":"build-2784847793"},"reference-count":145,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2020,3,1]],"date-time":"2020-03-01T00:00:00Z","timestamp":1583020800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2020,3,1]],"date-time":"2020-03-01T00:00:00Z","timestamp":1583020800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2019,7,25]],"date-time":"2019-07-25T00:00:00Z","timestamp":1564012800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100014461","name":"NIHR Biomedical Research Centre, Royal Marsden NHS Foundation Trust\/Institute of Cancer Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014461","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000765","name":"University College London","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000765","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81202316"],"award-info":[{"award-number":["81202316"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003784","name":"Great Ormond Street Hospital for Children","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003784","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010269","name":"Wellcome Trust","doi-asserted-by":"publisher","award":["104807\/Z\/14\/Z"],"award-info":[{"award-number":["104807\/Z\/14\/Z"]}],"id":[{"id":"10.13039\/100010269","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Genes &amp; Diseases"],"published-print":{"date-parts":[[2020,3]]},"DOI":"10.1016\/j.gendis.2019.07.007","type":"journal-article","created":{"date-parts":[[2019,7,30]],"date-time":"2019-07-30T02:22:31Z","timestamp":1564453351000},"page":"38-51","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":32,"title":["Gene therapy and genome editing for primary immunodeficiency diseases"],"prefix":"10.1016","volume":"7","author":[{"given":"Zhi-Yong","family":"Zhang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian J.","family":"Thrasher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fang","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib1","doi-asserted-by":"crossref","DOI":"10.1016\/j.jaci.2010.06.015","article-title":"Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?","volume":"126","author":"Gennery","year":"2010","journal-title":"J Allergy Clin Immunol"},{"issue":"5466","key":"10.1016\/j.gendis.2019.07.007_bib2","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"},{"issue":"5644","key":"10.1016\/j.gendis.2019.07.007_bib3","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1126\/science.1088547","article-title":"LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1","volume":"302","author":"Hacein-Bey-Abina","year":"2003","journal-title":"Science"},{"issue":"15","key":"10.1016\/j.gendis.2019.07.007_bib4","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"},{"issue":"335","key":"10.1016\/j.gendis.2019.07.007_bib5","doi-asserted-by":"crossref","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"},{"issue":"11","key":"10.1016\/j.gendis.2019.07.007_bib6","doi-asserted-by":"crossref","first-page":"1327","DOI":"10.1182\/blood-2017-04-777136","article-title":"Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult","volume":"130","author":"Morris","year":"2017","journal-title":"Blood"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib7","doi-asserted-by":"crossref","first-page":"1132","DOI":"10.1016\/j.ymthe.2017.03.018","article-title":"Evolving gene therapy in primary immunodeficiency","volume":"25","author":"Thrasher","year":"2017","journal-title":"Mol Ther"},{"issue":"6148","key":"10.1016\/j.gendis.2019.07.007_bib8","doi-asserted-by":"crossref","first-page":"1233151","DOI":"10.1126\/science.1233151","article-title":"Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome","volume":"341","author":"Aiuti","year":"2013","journal-title":"Science"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib9","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1007\/s12185-016-2017-z","article-title":"Gene editing and its application for hematological diseases","volume":"104","author":"Osborn","year":"2016","journal-title":"Int J Hematol"},{"issue":"10","key":"10.1016\/j.gendis.2019.07.007_bib10","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1089\/hum.2016.107","article-title":"Gene editing of human hematopoietic stem and progenitor cells: promise and potential hurdles","volume":"27","author":"Yu","year":"2016","journal-title":"Hum Gene Ther"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib11","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1007\/s12033-018-0072-9","article-title":"Genome editing in stem cells for disease therapeutics","volume":"60","author":"Song","year":"2018","journal-title":"Mol Biotechnol"},{"issue":"5235","key":"10.1016\/j.gendis.2019.07.007_bib12","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1126\/science.270.5235.475","article-title":"T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years","volume":"270","author":"Blaese","year":"1995","journal-title":"Science"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib13","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1182\/blood.V91.1.30","article-title":"Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency","volume":"91","author":"Onodera","year":"1998","journal-title":"Blood"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib14","first-page":"291","article-title":"Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy","volume":"35","author":"Hershfield","year":"1998","journal-title":"Semin Hematol"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib15","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1007\/s10875-017-0373-y","article-title":"How we manage adenosine deaminase-deficient severe combined immune deficiency (ADA SCID)","volume":"37","author":"Kohn","year":"2017","journal-title":"J Clin Immunol"},{"issue":"17","key":"10.1016\/j.gendis.2019.07.007_bib16","doi-asserted-by":"crossref","first-page":"3524","DOI":"10.1182\/blood-2009-06-189209","article-title":"How I treat ADA deficiency","volume":"114","author":"Gaspar","year":"2009","journal-title":"Blood"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib17","doi-asserted-by":"crossref","first-page":"434","DOI":"10.1056\/NEJMoa1401177","article-title":"Transplantation outcomes for severe combined immunodeficiency, 2000\u20132009","volume":"371","author":"Pai","year":"2014","journal-title":"N Engl J Med"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib18","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1016\/j.clim.2005.07.006","article-title":"Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID)","volume":"117","author":"Chan","year":"2005","journal-title":"Clin Immunol"},{"issue":"5235","key":"10.1016\/j.gendis.2019.07.007_bib19","doi-asserted-by":"crossref","first-page":"470","DOI":"10.1126\/science.270.5235.470","article-title":"Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients","volume":"270","author":"Bordignon","year":"1995","journal-title":"Science"},{"issue":"10","key":"10.1016\/j.gendis.2019.07.007_bib20","doi-asserted-by":"crossref","first-page":"1017","DOI":"10.1038\/nm1095-1017","article-title":"Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency","volume":"1","author":"Kohn","year":"1995","journal-title":"Nat Med"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib21","first-page":"179","article-title":"Bone marrow gene transfer in three patients with adenosine deaminase deficiency","volume":"3","author":"Hoogerbrugge","year":"1996","journal-title":"Gene Ther"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib22","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1182\/blood-2016-01-688226","article-title":"Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency","volume":"128","author":"Cicalese","year":"2016","journal-title":"Blood"},{"issue":"97","key":"10.1016\/j.gendis.2019.07.007_bib23","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3002716","article-title":"Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction","volume":"3","author":"Gaspar","year":"2011","journal-title":"Sci Transl Med"},{"issue":"18","key":"10.1016\/j.gendis.2019.07.007_bib24","doi-asserted-by":"crossref","first-page":"3635","DOI":"10.1182\/blood-2012-02-400937","article-title":"Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans","volume":"120","author":"Candotti","year":"2012","journal-title":"Blood"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib25","doi-asserted-by":"crossref","first-page":"1689","DOI":"10.1172\/JCI90367","article-title":"Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency","volume":"127","author":"Shaw","year":"2017","journal-title":"J Clin Invest"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib26","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1007\/s10875-015-0157-1","article-title":"Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning","volume":"35","author":"Otsu","year":"2015","journal-title":"J Clin Immunol"},{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib27","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1038\/mt.2013.265","article-title":"Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency","volume":"22","author":"Carbonaro","year":"2014","journal-title":"Mol Ther"},{"issue":"6304","key":"10.1016\/j.gendis.2019.07.007_bib28","doi-asserted-by":"crossref","first-page":"1101","DOI":"10.1126\/science.aah6192","article-title":"Lentiviral vectors, two decades later","volume":"353","author":"Naldini","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.gendis.2019.07.007_bib29","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1016\/j.coviro.2016.07.010","article-title":"Retroviral vector interactions with hematopoietic cells","volume":"21","author":"Everson","year":"2016","journal-title":"Curr Opin Virol"},{"issue":"18","key":"10.1016\/j.gendis.2019.07.007_bib30","doi-asserted-by":"crossref","first-page":"3677","DOI":"10.1182\/blood-2012-02-408591","article-title":"Gene therapy\/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction","volume":"120","author":"Carbonaro","year":"2012","journal-title":"Blood"},{"issue":"19","key":"10.1016\/j.gendis.2019.07.007_bib31","doi-asserted-by":"crossref","first-page":"2624","DOI":"10.1182\/blood-2016-12-756734","article-title":"Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients","volume":"129","author":"Cooper","year":"2017","journal-title":"Blood"},{"key":"10.1016\/j.gendis.2019.07.007_bib32","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.omtn.2016.11.011","article-title":"Limiting thymic precursor supply increases the risk of lymphoid malignancy in murine X-linked severe combined immunodeficiency","volume":"6","author":"Ginn","year":"2017","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.gendis.2019.07.007_bib33","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1146\/annurev.immunol.14.1.179","article-title":"The interleukin-2 receptor gamma chain: its role in the multiple cytokine receptor complexes and T cell development in XSCID","volume":"14","author":"Sugamura","year":"1996","journal-title":"Annu Rev Immunol"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib34","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1016\/0092-8674(93)90167-O","article-title":"Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans","volume":"73","author":"Noguchi","year":"1993","journal-title":"Cell"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib35","doi-asserted-by":"crossref","first-page":"274","DOI":"10.1073\/pnas.97.1.274","article-title":"Diversity, functionality, and stability of the T cell repertoire derived in\u00a0vivo from a single human T cell precursor","volume":"97","author":"Bousso","year":"2000","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"16","key":"10.1016\/j.gendis.2019.07.007_bib36","doi-asserted-by":"crossref","first-page":"1185","DOI":"10.1056\/NEJMoa012616","article-title":"Sustained correction of X-linked severe combined immunodeficiency by ex\u00a0vivo gene therapy","volume":"346","author":"Hacein-Bey-Abina","year":"2002","journal-title":"N Engl J Med"},{"issue":"9452","key":"10.1016\/j.gendis.2019.07.007_bib37","doi-asserted-by":"crossref","first-page":"2181","DOI":"10.1016\/S0140-6736(04)17590-9","article-title":"Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector","volume":"364","author":"Gaspar","year":"2004","journal-title":"Lancet"},{"issue":"9","key":"10.1016\/j.gendis.2019.07.007_bib38","doi-asserted-by":"crossref","first-page":"3143","DOI":"10.1172\/JCI35798","article-title":"Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients","volume":"118","author":"Howe","year":"2008","journal-title":"J Clin Invest"},{"issue":"9","key":"10.1016\/j.gendis.2019.07.007_bib39","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 Clin Invest"},{"issue":"11","key":"10.1016\/j.gendis.2019.07.007_bib40","doi-asserted-by":"crossref","first-page":"4255","DOI":"10.1182\/blood-2004-12-4837","article-title":"Failure of SCID-X1 gene therapy in older patients","volume":"105","author":"Thrasher","year":"2005","journal-title":"Blood"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib41","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1182\/blood-2006-11-058933","article-title":"Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency","volume":"110","author":"Chinen","year":"2007","journal-title":"Blood"},{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib42","doi-asserted-by":"crossref","first-page":"590","DOI":"10.1038\/sj.mt.6300393","article-title":"Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency","volume":"16","author":"Thornhill","year":"2008","journal-title":"Mol Ther"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib43","doi-asserted-by":"crossref","first-page":"718","DOI":"10.1038\/mt.2008.5","article-title":"Physiological promoters reduce the genotoxic risk of integrating gene vectors","volume":"16","author":"Zychlinski","year":"2008","journal-title":"Mol Ther"},{"issue":"15","key":"10.1016\/j.gendis.2019.07.007_bib44","doi-asserted-by":"crossref","first-page":"1407","DOI":"10.1056\/NEJMoa1404588","article-title":"A modified gamma-retrovirus vector for X-linked severe combined immunodeficiency","volume":"371","author":"Hacein-Bey-Abina","year":"2014","journal-title":"N Engl J Med"},{"issue":"9","key":"10.1016\/j.gendis.2019.07.007_bib45","doi-asserted-by":"crossref","first-page":"987","DOI":"10.1182\/bloodadvances.2018016451","article-title":"Rapid immune reconstitution of SCID-X1 canines after G-CSF\/AMD3100 mobilization and in\u00a0vivo gene therapy","volume":"2","author":"Humbert","year":"2018","journal-title":"Blood Adv"},{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib46","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1128\/br.39.3.169-185.1975","article-title":"The foamy viruses","volume":"39","author":"Hooks","year":"1975","journal-title":"Bacteriol Rev"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib47","doi-asserted-by":"crossref","first-page":"1498","DOI":"10.1073\/pnas.0510046103","article-title":"Foamy virus vector integration sites in normal human cells","volume":"103","author":"Trobridge","year":"2006","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.gendis.2019.07.007_bib48","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1146\/annurev.immunol.23.021704.115653","article-title":"How neutrophils kill microbes","volume":"23","author":"Segal","year":"2005","journal-title":"Annu Rev Immunol"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib49","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1093\/bmb\/ldw009","article-title":"Chronic granulomatous disease","volume":"118","author":"Roos","year":"2016","journal-title":"Br Med Bull"},{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib50","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1097\/00005792-200005000-00003","article-title":"Chronic granulomatous disease. Report on a national registry of 368 patients","volume":"79","author":"Winkelstein","year":"2000","journal-title":"Medicine (Baltim)"},{"issue":"13","key":"10.1016\/j.gendis.2019.07.007_bib51","doi-asserted-by":"crossref","first-page":"4344","DOI":"10.1182\/blood-2002-02-0583","article-title":"Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985\u20132000","volume":"100","author":"Seger","year":"2002","journal-title":"Blood"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib52","doi-asserted-by":"crossref","first-page":"28","DOI":"10.1038\/mt.2010.232","article-title":"Gene therapy of chronic granulomatous disease: the engraftment dilemma","volume":"19","author":"Grez","year":"2011","journal-title":"Mol Ther"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib53","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1016\/j.jaci.2015.11.028","article-title":"Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions","volume":"138","author":"Weisser","year":"2016","journal-title":"J Allergy Clin Immunol"},{"issue":"22","key":"10.1016\/j.gendis.2019.07.007_bib54","doi-asserted-by":"crossref","first-page":"12133","DOI":"10.1073\/pnas.94.22.12133","article-title":"Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease","volume":"94","author":"Malech","year":"1997","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib55","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1038\/nm1393","article-title":"Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1","volume":"12","author":"Ott","year":"2006","journal-title":"Nat Med"},{"issue":"11","key":"10.1016\/j.gendis.2019.07.007_bib56","doi-asserted-by":"crossref","first-page":"2092","DOI":"10.1038\/mt.2011.166","article-title":"Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I\/II trial","volume":"19","author":"Kang","year":"2011","journal-title":"Mol Ther"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib57","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1182\/blood-2009-05-222760","article-title":"Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils","volume":"115","author":"Kang","year":"2010","journal-title":"Blood"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib58","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1038\/nm.2088","article-title":"Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease","volume":"16","author":"Stein","year":"2010","journal-title":"Nat Med"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib59","doi-asserted-by":"crossref","first-page":"416","DOI":"10.2174\/1566523215666150515145255","article-title":"Successful combination of sequential gene therapy and rescue allo-HSCT in two children with X-CGD - importance of timing","volume":"15","author":"Siler","year":"2015","journal-title":"Curr Gene Ther"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib60","doi-asserted-by":"crossref","first-page":"122","DOI":"10.1038\/mt.2010.226","article-title":"Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells","volume":"19","author":"Santilli","year":"2011","journal-title":"Mol Ther"},{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib61","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1038\/nri2724","article-title":"WASP: a key immunological multitasker","volume":"10","author":"Thrasher","year":"2010","journal-title":"Nat Rev Immunol"},{"issue":"25","key":"10.1016\/j.gendis.2019.07.007_bib62","doi-asserted-by":"crossref","first-page":"6288","DOI":"10.1182\/blood-2008-12-115253","article-title":"Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome","volume":"113","author":"Bosticardo","year":"2009","journal-title":"Blood"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib63","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1097\/MOH.0b013e3282f30448","article-title":"Wiskott-Aldrich syndrome","volume":"15","author":"Notarangelo","year":"2008","journal-title":"Curr Opin Hematol"},{"issue":"6","key":"10.1016\/j.gendis.2019.07.007_bib64","doi-asserted-by":"crossref","first-page":"1675","DOI":"10.1182\/blood-2010-11-319376","article-title":"Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980\u20132009: an international collaborative study","volume":"118","author":"Moratto","year":"2011","journal-title":"Blood"},{"issue":"20","key":"10.1016\/j.gendis.2019.07.007_bib65","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 Engl J Med"},{"issue":"227","key":"10.1016\/j.gendis.2019.07.007_bib66","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3007280","article-title":"Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity","volume":"6","author":"Braun","year":"2014","journal-title":"Sci Transl Med"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib67","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1016\/j.hoc.2017.05.003","article-title":"Gene therapy approaches to immunodeficiency","volume":"31","author":"Ghosh","year":"2017","journal-title":"Hematol Oncol Clin N Am"},{"issue":"7","key":"10.1016\/j.gendis.2019.07.007_bib68","doi-asserted-by":"crossref","first-page":"738","DOI":"10.1038\/nbt.3584","article-title":"Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin","volume":"34","author":"Palchaudhuri","year":"2016","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.gendis.2019.07.007_bib69","doi-asserted-by":"crossref","first-page":"308","DOI":"10.1196\/annals.1345.007","article-title":"A phase I\/II clinical trial of beta-globin gene therapy for beta-thalassemia","volume":"1054","author":"Bank","year":"2005","journal-title":"Ann N Y Acad Sci"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib70","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1038\/nbt.3062","article-title":"Genomic discovery of potent chromatin insulators for human gene therapy","volume":"33","author":"Liu","year":"2015","journal-title":"Nat Biotechnol"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib71","doi-asserted-by":"crossref","first-page":"10213","DOI":"10.1038\/s41598-017-10222-3","article-title":"Characterization of a core region in the A2UCOE that confers effective anti-silencing activity","volume":"7","author":"Zhang","year":"2017","journal-title":"Sci Rep"},{"key":"10.1016\/j.gendis.2019.07.007_bib72","doi-asserted-by":"crossref","first-page":"486","DOI":"10.1016\/j.biomaterials.2018.11.006","article-title":"Sustained and regulated gene expression by Tet-inducible \"all-in-one\" retroviral vectors containing the HNRPA2B1-CBX3 UCOE((R))","volume":"192","author":"Cullmann","year":"2019","journal-title":"Biomaterials"},{"issue":"6","key":"10.1016\/j.gendis.2019.07.007_bib73","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1016\/j.stem.2018.10.008","article-title":"Cyclosporine H overcomes innate immune restrictions to improve lentiviral transduction and gene editing in human hematopoietic stem cells","volume":"23","author":"Petrillo","year":"2018","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.gendis.2019.07.007_bib74","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1016\/j.omtm.2018.08.002","article-title":"A nontoxic transduction enhancer enables highly efficient lentiviral transduction of primary murine T cells and hematopoietic stem cells","volume":"10","author":"Delville","year":"2018","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib75","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1038\/nrm2351","article-title":"Regulation of DNA repair throughout the cell cycle","volume":"9","author":"Branzei","year":"2008","journal-title":"Nat Rev Mol Cell Biol"},{"issue":"9","key":"10.1016\/j.gendis.2019.07.007_bib76","doi-asserted-by":"crossref","first-page":"636","DOI":"10.1038\/nrg2842","article-title":"Genome editing with engineered zinc finger nucleases","volume":"11","author":"Urnov","year":"2010","journal-title":"Nat Rev Genet"},{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib77","doi-asserted-by":"crossref","first-page":"1156","DOI":"10.1073\/pnas.93.3.1156","article-title":"Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain","volume":"93","author":"Kim","year":"1996","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"14","key":"10.1016\/j.gendis.2019.07.007_bib78","doi-asserted-by":"crossref","first-page":"6315","DOI":"10.1093\/nar\/gkr188","article-title":"Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes","volume":"39","author":"Li","year":"2011","journal-title":"Nucleic Acids Res"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib79","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1038\/nbt.1755","article-title":"A TALE nuclease architecture for efficient genome editing","volume":"29","author":"Miller","year":"2011","journal-title":"Nat Biotechnol"},{"issue":"6","key":"10.1016\/j.gendis.2019.07.007_bib80","doi-asserted-by":"crossref","first-page":"1262","DOI":"10.1016\/j.cell.2014.05.010","article-title":"Development and applications of CRISPR-Cas9 for genome engineering","volume":"157","author":"Hsu","year":"2014","journal-title":"Cell"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib81","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1038\/nbt.2842","article-title":"CRISPR-Cas systems for editing, regulating and targeting genomes","volume":"32","author":"Sander","year":"2014","journal-title":"Nat Biotechnol"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib82","doi-asserted-by":"crossref","first-page":"300","DOI":"10.1038\/nrg.2016.28","article-title":"Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases","volume":"17","author":"Tsai","year":"2016","journal-title":"Nat Rev Genet"},{"issue":"7385","key":"10.1016\/j.gendis.2019.07.007_bib83","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1038\/nature10886","article-title":"RNA-guided genetic silencing systems in bacteria and archaea","volume":"482","author":"Wiedenheft","year":"2012","journal-title":"Nature"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib84","doi-asserted-by":"crossref","first-page":"582","DOI":"10.1134\/S0026893317040033","article-title":"CRISPR\/CAS9, the king of genome editing tools","volume":"51","author":"Bannikov","year":"2017","journal-title":"Mol Biol (Mosk)"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib85","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1139\/bcb-2016-0137","article-title":"Precision genome editing in the CRISPR era","volume":"95","author":"Salsman","year":"2017","journal-title":"Biochem Cell Biol"},{"key":"10.1016\/j.gendis.2019.07.007_bib86","article-title":"Highly efficient gene disruption of murine and human hematopoietic progenitor cells by CRISPR\/Cas9","volume":"134","author":"Brunetti","year":"2018","journal-title":"J Vis Exp"},{"key":"10.1016\/j.gendis.2019.07.007_bib87","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1016\/bs.pmbts.2017.05.003","article-title":"CRISPR\/Cas9-Enabled multiplex genome editing and its application","volume":"149","author":"Minkenberg","year":"2017","journal-title":"Prog Mol Biol Transl Sci"},{"issue":"7504","key":"10.1016\/j.gendis.2019.07.007_bib88","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"},{"issue":"5997","key":"10.1016\/j.gendis.2019.07.007_bib89","doi-asserted-by":"crossref","first-page":"1345","DOI":"10.1126\/science.1191536","article-title":"Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells","volume":"329","author":"Boitano","year":"2010","journal-title":"Science"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib90","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1016\/j.stem.2011.02.003","article-title":"Prostaglandin E2 enhances human cord blood stem cell xenotransplants and shows long-term safety in preclinical nonhuman primate transplant models","volume":"8","author":"Goessling","year":"2011","journal-title":"Cell Stem Cell"},{"issue":"6203","key":"10.1016\/j.gendis.2019.07.007_bib91","doi-asserted-by":"crossref","first-page":"1509","DOI":"10.1126\/science.1256337","article-title":"Cord blood expansion. Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal","volume":"345","author":"Fares","year":"2014","journal-title":"Science"},{"key":"10.1016\/j.gendis.2019.07.007_bib92","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/j.omtn.2018.04.017","article-title":"Priming human repopulating hematopoietic stem and progenitor cells for Cas9\/sgRNA gene targeting","volume":"12","author":"Charlesworth","year":"2018","journal-title":"Mol Ther Nucleic Acids"},{"issue":"10","key":"10.1016\/j.gendis.2019.07.007_bib93","doi-asserted-by":"crossref","first-page":"1852","DOI":"10.1002\/sctm.17-0048","article-title":"Brief report: a differential transcriptomic profile of ex\u00a0vivo expanded adult human hematopoietic stem cells empowers them for engraftment better than their surface phenotype","volume":"6","author":"Psatha","year":"2017","journal-title":"Stem Cells Transl Med"},{"key":"10.1016\/j.gendis.2019.07.007_bib94","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 beta-thalassemia major","volume":"10","author":"Psatha","year":"2018","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"6121","key":"10.1016\/j.gendis.2019.07.007_bib95","doi-asserted-by":"crossref","first-page":"786","DOI":"10.1126\/science.1232458","article-title":"Cyclic GMP-AMP synthase is a cytosolic DNA sensor that activates the type I interferon pathway","volume":"339","author":"Sun","year":"2013","journal-title":"Science"},{"issue":"12","key":"10.1016\/j.gendis.2019.07.007_bib96","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.gendis.2019.07.007_bib97","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1016\/j.jbiotec.2015.04.024","article-title":"Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection","volume":"208","author":"Liang","year":"2015","journal-title":"J Biotechnol"},{"issue":"4","key":"10.1016\/j.gendis.2019.07.007_bib98","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"},{"issue":"411","key":"10.1016\/j.gendis.2019.07.007_bib99","doi-asserted-by":"crossref","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"},{"issue":"9","key":"10.1016\/j.gendis.2019.07.007_bib100","doi-asserted-by":"crossref","first-page":"2606","DOI":"10.1016\/j.celrep.2018.04.103","article-title":"Site-specific gene editing of human hematopoietic stem cells for X-linked hyper-IgM syndrome","volume":"23","author":"Kuo","year":"2018","journal-title":"Cell Rep"},{"issue":"7629","key":"10.1016\/j.gendis.2019.07.007_bib101","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1038\/nature20134","article-title":"CRISPR\/Cas9 beta-globin gene targeting in human haematopoietic stem cells","volume":"539","author":"Dever","year":"2016","journal-title":"Nature"},{"issue":"8","key":"10.1016\/j.gendis.2019.07.007_bib102","doi-asserted-by":"crossref","first-page":"1216","DOI":"10.1038\/s41591-018-0137-0","article-title":"A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells","volume":"24","author":"Vakulskas","year":"2018","journal-title":"Nat Med"},{"key":"10.1016\/j.gendis.2019.07.007_bib103","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.27873","article-title":"Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR\/Cas9 and AAV6","volume":"6","author":"Bak","year":"2017","journal-title":"Elife"},{"issue":"360","key":"10.1016\/j.gendis.2019.07.007_bib104","doi-asserted-by":"crossref","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"},{"issue":"372","key":"10.1016\/j.gendis.2019.07.007_bib105","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aah3480","article-title":"CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease","volume":"9","author":"De Ravin","year":"2017","journal-title":"Sci Transl Med"},{"issue":"9","key":"10.1016\/j.gendis.2019.07.007_bib106","doi-asserted-by":"crossref","first-page":"1705","DOI":"10.1038\/mt.2013.106","article-title":"Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus","volume":"21","author":"Joglekar","year":"2013","journal-title":"Mol Ther"},{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib107","doi-asserted-by":"crossref","first-page":"207","DOI":"10.2174\/1566523216666160602213738","article-title":"AAV vectorization of DSB-mediated gene editing technologies","volume":"16","author":"Moser","year":"2016","journal-title":"Curr Gene Ther"},{"issue":"7534","key":"10.1016\/j.gendis.2019.07.007_bib108","doi-asserted-by":"crossref","first-page":"360","DOI":"10.1038\/nature13864","article-title":"Promoterless gene targeting without nucleases ameliorates haemophilia B in mice","volume":"517","author":"Barzel","year":"2015","journal-title":"Nature"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib109","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1038\/nrg2988","article-title":"Therapeutic in\u00a0vivo gene transfer for genetic disease using AAV: progress and challenges","volume":"12","author":"Mingozzi","year":"2011","journal-title":"Nat Rev Genet"},{"issue":"3","key":"10.1016\/j.gendis.2019.07.007_bib110","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1016\/j.celrep.2017.06.064","article-title":"CRISPR-mediated integration of large gene cassettes using AAV donor vectors","volume":"20","author":"Bak","year":"2017","journal-title":"Cell Rep"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib111","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.gendis.2019.07.007_bib112","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1146\/annurev-genet-051710-150955","article-title":"Regulation of homologous recombination in eukaryotes","volume":"44","author":"Heyer","year":"2010","journal-title":"Annu Rev Genet"},{"issue":"6","key":"10.1016\/j.gendis.2019.07.007_bib113","doi-asserted-by":"crossref","first-page":"1555","DOI":"10.1016\/j.celrep.2016.01.019","article-title":"Post-translational regulation of Cas9 during G1 enhances homology-directed repair","volume":"14","author":"Gutschner","year":"2016","journal-title":"Cell Rep"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib114","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1002\/stem.2935","article-title":"Improving gene editing outcomes in human hematopoietic stem and progenitor cells by temporal control of DNA repair","volume":"37","author":"Lomova","year":"2019","journal-title":"Stem Cells"},{"issue":"28","key":"10.1016\/j.gendis.2019.07.007_bib115","doi-asserted-by":"crossref","first-page":"10660","DOI":"10.1073\/pnas.0600447103","article-title":"Selective small-molecule inhibitor reveals critical mitotic functions of human CDK1","volume":"103","author":"Vassilev","year":"2006","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib116","doi-asserted-by":"crossref","first-page":"538","DOI":"10.1038\/nbt.3190","article-title":"Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining","volume":"33","author":"Maruyama","year":"2015","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.gendis.2019.07.007_bib117","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1186\/s13578-018-0200-z","article-title":"Ligase IV inhibitor SCR7 enhances gene editing directed by CRISPR-Cas9 and ssODN in human cancer cells","volume":"8","author":"Hu","year":"2018","journal-title":"Cell Biosci"},{"issue":"6","key":"10.1016\/j.gendis.2019.07.007_bib118","doi-asserted-by":"crossref","first-page":"e42","DOI":"10.1093\/nar\/gkt1326","article-title":"An online bioinformatics tool predicts zinc finger and TALE nuclease off-target cleavage","volume":"42","author":"Fine","year":"2014","journal-title":"Nucleic Acids Res"},{"issue":"9","key":"10.1016\/j.gendis.2019.07.007_bib119","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"},{"issue":"9","key":"10.1016\/j.gendis.2019.07.007_bib120","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1038\/nbt.1948","article-title":"An unbiased genome-wide analysis of zinc-finger nuclease specificity","volume":"29","author":"Gabriel","year":"2011","journal-title":"Nat Biotechnol"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib121","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/j.stem.2014.04.020","article-title":"Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing","volume":"15","author":"Veres","year":"2014","journal-title":"Cell Stem Cell"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib122","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1016\/j.stem.2014.06.017","article-title":"What's changed with genome editing?","volume":"15","author":"Tsai","year":"2014","journal-title":"Cell Stem Cell"},{"issue":"7","key":"10.1016\/j.gendis.2019.07.007_bib123","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":"Hutter","year":"2009","journal-title":"N Engl J Med"},{"issue":"8","key":"10.1016\/j.gendis.2019.07.007_bib124","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"},{"issue":"6","key":"10.1016\/j.gendis.2019.07.007_bib125","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.gendis.2019.07.007_bib126","doi-asserted-by":"crossref","first-page":"16067","DOI":"10.1038\/mtm.2016.67","article-title":"Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem\/progenitor cells","volume":"3","author":"DiGiusto","year":"2016","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"8","key":"10.1016\/j.gendis.2019.07.007_bib127","doi-asserted-by":"crossref","first-page":"1782","DOI":"10.1016\/j.ymthe.2017.04.027","article-title":"CRISPR\/Cas9-Mediated CCR5 ablation in human hematopoietic stem\/progenitor cells confers HIV-1 resistance in\u00a0vivo","volume":"25","author":"Xu","year":"2017","journal-title":"Mol Ther"},{"issue":"13","key":"10.1016\/j.gendis.2019.07.007_bib128","doi-asserted-by":"crossref","first-page":"1021","DOI":"10.1056\/NEJM199904013401307","article-title":"Management of sickle cell disease","volume":"340","author":"Steinberg","year":"1999","journal-title":"N Engl J Med"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib129","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1182\/blood.V85.1.43.bloodjournal85143","article-title":"Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial","volume":"85","author":"Collins","year":"1995","journal-title":"Blood"},{"key":"10.1016\/j.gendis.2019.07.007_bib130","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1016\/j.gde.2015.08.001","article-title":"Hemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin","volume":"33","author":"Bauer","year":"2015","journal-title":"Curr Opin Genet Dev"},{"key":"10.1016\/j.gendis.2019.07.007_bib131","doi-asserted-by":"crossref","first-page":"e351","DOI":"10.1038\/mtna.2016.52","article-title":"Reactivating fetal hemoglobin expression in human adult erythroblasts through BCL11A knockdown using targeted endonucleases","volume":"5","author":"Bjurstrom","year":"2016","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.gendis.2019.07.007_bib132","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"},{"issue":"1 Pt 1","key":"10.1016\/j.gendis.2019.07.007_bib133","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/S0022-3476(97)70123-9","article-title":"Clinical spectrum of X-linked hyper-IgM syndrome","volume":"131","author":"Levy","year":"1997","journal-title":"J Pediatr"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib134","doi-asserted-by":"crossref","first-page":"977","DOI":"10.4049\/jimmunol.158.2.977","article-title":"Cholangiopathy and tumors of the pancreas, liver, and biliary tree in boys with X-linked immunodeficiency with hyper-IgM","volume":"158","author":"Hayward","year":"1997","journal-title":"J Immunol"},{"issue":"11","key":"10.1016\/j.gendis.2019.07.007_bib135","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1038\/3233","article-title":"Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice","volume":"4","author":"Brown","year":"1998","journal-title":"Nat Med"},{"issue":"10","key":"10.1016\/j.gendis.2019.07.007_bib136","doi-asserted-by":"crossref","first-page":"1299","DOI":"10.1038\/sj.cgt.7700232","article-title":"Lymphoid abnormalities in CD40 ligand transgenic mice suggest the need for tight regulation in gene therapy approaches to hyper immunoglobulin M (IgM) syndrome","volume":"7","author":"Sacco","year":"2000","journal-title":"Cancer Gene Ther"},{"issue":"21","key":"10.1016\/j.gendis.2019.07.007_bib137","doi-asserted-by":"crossref","first-page":"2513","DOI":"10.1182\/blood-2015-11-683235","article-title":"Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome","volume":"127","author":"Hubbard","year":"2016","journal-title":"Blood"},{"issue":"2","key":"10.1016\/j.gendis.2019.07.007_bib138","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1038\/nbt.3117","article-title":"GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases","volume":"33","author":"Tsai","year":"2015","journal-title":"Nat Biotechnol"},{"issue":"7","key":"10.1016\/j.gendis.2019.07.007_bib139","doi-asserted-by":"crossref","first-page":"927","DOI":"10.1038\/s41591-018-0049-z","article-title":"CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response","volume":"24","author":"Haapaniemi","year":"2018","journal-title":"Nat Med"},{"issue":"1","key":"10.1016\/j.gendis.2019.07.007_bib140","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1111\/nyas.12986","article-title":"Regulation of hematopoietic stem cell integrity through p53 and its related factors","volume":"1370","author":"Yamashita","year":"2016","journal-title":"Ann N Y Acad Sci"},{"issue":"8","key":"10.1016\/j.gendis.2019.07.007_bib141","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"},{"issue":"6","key":"10.1016\/j.gendis.2019.07.007_bib142","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1038\/nmeth.4278","article-title":"CIRCLE-seq: a highly sensitive in\u00a0vitro screen for genome-wide CRISPR-Cas9 nuclease off-targets","volume":"14","author":"Tsai","year":"2017","journal-title":"Nat Methods"},{"issue":"7587","key":"10.1016\/j.gendis.2019.07.007_bib143","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"},{"issue":"5","key":"10.1016\/j.gendis.2019.07.007_bib144","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1038\/nmeth.3330","article-title":"Improved specificity of TALE-based genome editing using an expanded RVD repertoire","volume":"12","author":"Miller","year":"2015","journal-title":"Nat Methods"},{"issue":"8","key":"10.1016\/j.gendis.2019.07.007_bib145","doi-asserted-by":"crossref","first-page":"863","DOI":"10.1038\/nbt.3609","article-title":"Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells","volume":"34","author":"Kim","year":"2016","journal-title":"Nat Biotechnol"}],"container-title":["Genes &amp; Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2352304219300510?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2352304219300510?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T17:25:26Z","timestamp":1762449926000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2352304219300510"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,3]]},"references-count":145,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2020,3]]}},"alternative-id":["S2352304219300510"],"URL":"https:\/\/doi.org\/10.1016\/j.gendis.2019.07.007","relation":{},"ISSN":["2352-3042"],"issn-type":[{"value":"2352-3042","type":"print"}],"subject":[],"published":{"date-parts":[[2020,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Gene therapy and genome editing for primary immunodeficiency diseases","name":"articletitle","label":"Article Title"},{"value":"Genes & Diseases","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.gendis.2019.07.007","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2020 Chongqing Medical University. Production and hosting by Elsevier B.V.","name":"copyright","label":"Copyright"}]}}