Journal
VISION RESEARCH
Volume 48, Issue 3, Pages 353-359Publisher
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.visres.2007.07.027
Keywords
AAV; gene therapy; retina
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Funding
- NEI NIH HHS [R01 EY015136, 1R01EY015136-01] Funding Source: Medline
- Telethon [TGM06S01, TGM06C03] Funding Source: Medline
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Gene therapy represents a promising therapeutic option for many inherited and acquired retinal diseases. Recombinant adeno-associated viral vectors (AAV) are the most efficient tools to transfer genes in vivo to the retina. The recent identification of dozens of novel AAV serotypes enormously expands on the versatility of AAV as vector system for in vivo somatic gene transfer. The results from the forthcoming trials with AAV in the retina of patients with Leber Congenital Amaurosis will be critical for the rapid development of AAV-based therapeutics for retinal diseases. (c) 2007 Elsevier Ltd. All rights reserved.
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