UMMS Affiliation

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

Publication Date


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.


gene therapy

Rights and Permissions

Copyright 2017 The Authors.

DOI of Published Version



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

Journal/Book/Conference Title

Clinical and translational science

Related Resources

Link to Article in PubMed

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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