IngentaConnect Adeno-Associated Viral Vectors for Retinal Gene Transfer and Trea...

Authors: Auricchio, Alberto¹; Rolling, Fabienne¹

Source: Current Gene Therapy, Volume 5, Number 3, June 2005 , pp. 339-348(10)

Publisher: Bentham Science Publishers

Abstract:

Retinal gene transfer holds big promises for the treatment of inherited and non-inherited blinding diseases, such as retinitis pigmentosa or age-related macular degeneration. Key to the development of successful gene-based therapies for the eye are efficient tools for retinal gene transfer. Vectors based on adeno-associated viruses (AAV) are able to transduce robustly and persistently different retinal cell types of animal models after a single intraocular administration. Recombinant AAV (rAAV) vectors are versatile gene transfer tools in that capsid proteins from dozens of AAV serotypes can be easily interchanged, resulting in the creation of recombinant vectors with unique transduction properties. This has allowed successful proof-of-principle studies using rAAV-mediated gene transfer to restore retinal morphology and function in small and large animal models of retinal diseases. In addition, gene delivery using rAAV vectors in the eye seems to have appropriate biosafety characteristics to rapidly move it from bench to bedside. All the above aspects will be reviewed and discussed in detail below.

Keywords: retina; gene transfer; raav; retinal degeneration; retinitis pigmentosa; neovascular diseases

Document Type: Review article

DOI: 10.2174/1566523054065020

Affiliations: 1: Laboratoire de Therapie Genique, INSERM U649, CHU-Hotel DIEU, Bat. J. Monnet, 30 Avenue J. Monnet, 44035 Nantes Cedex 01, France.

Your trusted access to this article has expired.

The full text electronic article is available for purchase. You will be able to download the full text electronic article after payment.

$55.10 plus tax Refund Policy

Adeno-Associated Viral Vectors for Retinal Gene Transfer and Treatment of Retinal Diseases