Versatility of AAV vectors for retinal gene transfer.

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.

• Authors: Enrico M Surace, Alberto Auricchio
• Journal: Vision research (Vision Res) Vol. 48 Issue 3 Pg. 353-9 (Feb 2008) ISSN: 0042-6989 [Print] England
• PMID: 17923143 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Review)

Topics

• Animals
• Dependovirus (genetics)
• Eye Diseases, Hereditary (therapy)
• Gene Transfer Techniques
• Genetic Therapy (methods)
• Genetic Vectors
• Humans
• Retinal Diseases (therapy)
• Transduction, Genetic