UMMS Affiliation

Department of Pediatrics, Division of Pediatric Pulmonology; Horae Gene Therapy Center



Document Type



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.

Rights and Permissions

Copyright 2017 The Authors. Citation: Clin Transl Sci. 2017 Apr 6. doi: 10.1111/cts.12466. Link to article on publisher's site

Related Resources

Link to Article in PubMed


gene therapy

PubMed ID


Creative Commons License

Creative Commons Attribution-Noncommercial 4.0 License
This work is licensed under a Creative Commons Attribution-Noncommercial 4.0 License



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