UMass Chan AffiliationsHorae Gene Therapy Center
Department of Pediatrics, Division of Pediatric Pulmonology
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AbstractIntroduction: 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.
SourceClin Transl Sci. 2017 Apr 6. doi: 10.1111/cts.12466. Link to article on publisher's site
Permanent Link to this Itemhttp://hdl.handle.net/20.500.14038/43554
Related ResourcesLink to Article in PubMed
RightsCopyright 2017 The Authors.