Article

Adeno-associated virus-vectored gene therapy for retinal disease.

Department of Ophthalmology, Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
Human Gene Therapy (impact factor: 4.22). 07/2005; 16(6):649-63. DOI:10.1089/hum.2005.16.649 pp.649-63
Source: PubMed

ABSTRACT Recombinant adeno-associated viral (AAV) vectors have become powerful gene delivery tools for the treatment of retinal degeneration in a variety of animal models that mimic corresponding human diseases. AAV vectors possess a number of features that render them ideally suited for retinal gene therapy, including a lack of pathogenicity, minimal immunogenicity, and the ability to transduce postmitotic cells in a stable and efficient manner. In the sheltered environment of the retina, AAV vectors are able to maintain high levels of transgene expression in the retinal pigmented epithelium (RPE), photoreceptors, or ganglion cells for long periods of time after a single treatment. Each cell type can be specifically targeted by choosing the appropriate combination of AAV serotype, promoter, and intraocular injection site. The focus of this review is on examples of AAV-mediated gene therapy in those animal models of inherited retinal degeneration caused by mutations directly affecting the interacting unit formed by photoreceptors and the RPE. In each case discussed, expression of the therapeutic gene resulted in significant recovery of retinal structure and/or visual function. Because of the key role of the vasculature in maintaining a healthy retina, a summary of AAV gene therapy applications in animal models of retinal neovascular diseases is also included.

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Data provided are for informational purposes only. Although carefully collected, accuracy cannot be guaranteed. The impact factor represents a rough estimation of the journal's impact factor and does not reflect the actual current impact factor. Publisher conditions are provided by RoMEO. Differing provisions from the publisher's actual policy or licence agreement may be applicable.

Keywords

AAV gene therapy applications
 
AAV vectors
 
AAV-mediated gene therapy
 
appropriate combination
 
ganglion cells
 
healthy retina
 
intraocular injection site
 
mimic corresponding human diseases
 
minimal immunogenicity
 
powerful gene delivery tools
 
Recombinant adeno-associated viral
 
retinal degeneration
 
retinal gene therapy
 
retinal neovascular diseases
 
retinal pigmented epithelium
 
retinal structure
 
single treatment
 
therapeutic gene
 
transduce postmitotic cells
 
transgene expression