UMMS Affiliation

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

Publication Date

2017-04-06

Document Type

Article

Disciplines

Genetics and Genomics | Therapeutics | Translational Medical Research

Abstract

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.

Keywords

gene therapy

Rights and Permissions

Copyright 2017 The Authors.

DOI of Published Version

10.1111/cts.12466

Source

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

28383804

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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