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Vision Res. 2008 Feb;48(3):353-9. Epub 2007 Oct 17.

Versatility of AAV vectors for retinal gene transfer.

Author information

1
Telethon Institute of Genetics and Medicine, Naples, Italy.

Abstract

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.

PMID:
17923143
DOI:
10.1016/j.visres.2007.07.027
[Indexed for MEDLINE]
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