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Review Article

Rescue of Sight by Gene Therapy—Closer than It May Appear

Pages 127-133 | Received 05 Apr 2007, Accepted 15 May 2007, Published online: 08 Jul 2009
 

Abstract

This review will cover the state of the field in retinal degeneration and gene therapy with a focus on the great strides that have been made in retina gene therapy. Topics ranging from the development of animal models to clinical trials (for the treatment of Leber congenital amaurosis, age-related macular degeneration, and retinoblastoma) will be discussed. In addition, the results of gene therapy studies targeting the photoreceptors will be presented. Finally, strategies and progress in overcoming the challenges of photoreceptor-directed gene therapy will be presented.

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