Department of Pediatrics, Division of Pediatric Pulmonology; Horae Gene Therapy Center
Genetics and Genomics | Therapeutics | Translational Medical Research
Introduction: Gene therapy has changed dramatically in the 28 years since the first human gene transfer experiment in 1989. Alipogene tiparvovec, GlyberaR®, a recombinant adeno-associated virus (rAAV) product for lipoprotein lipase deficiency, and Strimvelis®, a lentivirus vector for severe combined immune deficiency are approved in Europe. An rAAV2 product for a congenital form of blindness is currently under review in the United States, likely to be followed by numerous other gene therapies.
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DOI of Published Version
Clin Transl Sci. 2017 Apr 6. doi: 10.1111/cts.12466. Link to article on publisher's site
Clinical and translational science
Keeler, Allison M.; Elmallah, Mai K.; and Flotte, Terence R., "Gene Therapy 2017: Progress and Future Directions" (2017). Pediatric Publications and Presentations. 125.
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