{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T04:16:31Z","timestamp":1791346591871,"version":"4.3.1"},"reference-count":255,"publisher":"Wiley","issue":"24","license":[{"start":{"date-parts":[[2019,11,20]],"date-time":"2019-11-20T00:00:00Z","timestamp":1574208000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","award":["R01HL128288"],"award-info":[{"award-number":["R01HL128288"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","award":["R01HL141781"],"award-info":[{"award-number":["R01HL141781"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["febs.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["FEBS Letters"],"published-print":{"date-parts":[[2019,12]]},"abstract":"<jats:p>\n                    Genome editing of hematopoietic stem cells (HSCs) represents a therapeutic option for a number of hematological genetic diseases, as HSCs have the potential for self\u2010renewal and differentiation into all blood cell lineages. This review presents advances of genome editing in HSCs utilizing adenovirus vectors as delivery vehicles. We focus on capsid\u2010modified, helper\u2010dependent adenovirus vectors that are devoid of all viral genes and therefore exhibit an improved safety profile. We discuss HSC genome engineering for several inherited disorders and infectious diseases including hemoglobinopathies, Fanconi anemia, hemophilia, and HIV\u20101 infection by\n                    <jats:italic>ex\u00a0vivo<\/jats:italic>\n                    and\n                    <jats:italic>in\u00a0vivo<\/jats:italic>\n                    editing in transgenic mice, nonhuman primates, as well as in human CD34\n                    <jats:sup>+<\/jats:sup>\n                    cells. Mechanisms of therapeutic gene transfer including episomal expression of designer nucleases and base editors, transposase\u2010mediated random integration, and targeted homology\u2010directed repair triggered integration into selected genomic safe harbor loci are also reviewed.\n                  <\/jats:p>","DOI":"10.1002\/1873-3468.13668","type":"journal-article","created":{"date-parts":[[2019,11,9]],"date-time":"2019-11-09T13:04:56Z","timestamp":1573304696000},"page":"3623-3648","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":54,"title":["Adenovirus vectors in hematopoietic stem cell genome editing"],"prefix":"10.1002","volume":"593","author":[{"given":"Chang","family":"Li","sequence":"first","affiliation":[{"name":"Department of Medicine Division of Medical Genetics University of Washington Seattle WA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andr\u00e9","family":"Lieber","sequence":"additional","affiliation":[{"name":"Department of Medicine Division of Medical Genetics University of Washington Seattle WA USA"},{"name":"Department of Pathology University of Washington Seattle WA 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