{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T20:35:24Z","timestamp":1782506124156,"version":"3.54.5"},"reference-count":111,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2018,3,29]],"date-time":"2018-03-29T00:00:00Z","timestamp":1522281600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["faseb.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["The FASEB Journal"],"published-print":{"date-parts":[[2018,4]]},"abstract":"<jats:p>\n                    Gene therapy is emerging as a viable option for clinical therapy of monogenic disorders and other genetically defined diseases, with approved gene therapies available in Europe and newly approved gene therapies in the United States. In the past 10 years, gene therapy has moved from a distant possibility, even in the minds of much of the scientific community, to being widely realized as a valuable therapeutic tool with wide\u2010ranging potential. The U.S. Food and Drug Administration has recently approved Luxturna (Spark Therapeutics Inc, Philadelphia, PA, USA), a recombinant adeno\u2010associated virus (rAAV) 2 gene therapy for one type of Leber congenital amaurosis 2 (1, 2). The European Medicines Agency (EMA) has approved 3 recombinant viral vector products: Glybera (UniQure, Amsterdam, The Netherlands), an rAAV vector for lipoprotein lipase deficiency; Strimvelis (Glaxo Smith\u2010Kline, Brentford, United Kingdom), an\n                    <jats:italic>ex vivo<\/jats:italic>\n                    gammaretrovirus\u2010based therapy for patients with adenosine deaminase\u2010deficient severe combined immune deficiency (ADA\u2010SCID); and Kymriah (Novartis, Basel, Switzerland), an ex\n                    <jats:italic>vivo<\/jats:italic>\n                    lentivirus\u2010based therapy to engineer autologous chimeric antigen\u2010receptor T (CAR\u2010T) cells targeting CD19\u2013positive cells in acute lymphoblastic leukemia. These examples will be followed by the clinical approval of other gene therapy products as this field matures. In this review we provide an overview of the state of gene therapy by discussing where the field stands with respect to the different gene therapy vector platforms and the types of therapies that are available.\u2014 Gruntman, A. M., Flotte, T. R. The rapidly evolving state of gene therapy.\n                    <jats:italic>FASEB J.<\/jats:italic>\n                    32, 1733\u20131740 (2018).\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/www.fasebj.org\">www.fasebj.org<\/jats:ext-link>\n                  <\/jats:p>","DOI":"10.1096\/fj.201700982r","type":"journal-article","created":{"date-parts":[[2018,3,29]],"date-time":"2018-03-29T04:04:39Z","timestamp":1522296279000},"page":"1733-1740","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":41,"title":["The rapidly evolving state of gene therapy"],"prefix":"10.1096","volume":"32","author":[{"given":"Alisha M.","family":"Gruntman","sequence":"first","affiliation":[{"name":"Horae Gene Therapy Center Department of Pediatrics University of Massachusetts Medical School Worcester Massachusetts USA"},{"name":"Department of Clinical Sciences Cummings School of Veterinary Medicine at Tufts University North Grafton Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Terence R.","family":"Flotte","sequence":"additional","affiliation":[{"name":"Horae Gene Therapy Center Department of Pediatrics University of Massachusetts Medical School Worcester Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2018,3,29]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/mtm.2014.66"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1101\/cshperspect.a017285"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199008303230904"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.270.5235.475"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.3008226"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aac5415"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.78.9.5842"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.80.9.2519"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.80.15.4709"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.9.1.100"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra032207"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra032207"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1038\/ng0994-42"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1128\/jvi.69.4.2004-2015.1995"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.1997.8.10-1275"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.1997.8.1-45"},{"key":"e_1_2_6_18_1","first-page":"117","article-title":"Humoral and cellular immune responses of nonhuman primates to long\u2010term repeated lung exposure to Ad2\/CFTR\u20102","volume":"3","author":"Kaplan J. 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