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
Current Gene Therapy
Title: Adeno-Associated Viral Vectors for Retinal Gene Transfer and Treatment of Retinal Diseases
Volume: 5 Issue: 3
Author(s): Alberto Auricchio and Fabienne Rolling
Affiliation:
Keywords: retina, gene transfer, raav, retinal degeneration, retinitis pigmentosa, neovascular diseases
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.
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Cite this article as:
Auricchio Alberto and Rolling Fabienne, Adeno-Associated Viral Vectors for Retinal Gene Transfer and Treatment of Retinal Diseases, Current Gene Therapy 2005; 5 (3) . https://dx.doi.org/10.2174/1566523054065020
DOI https://dx.doi.org/10.2174/1566523054065020 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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