Rescue of sight by gene therapy - closer than it may appear.

Rex TS
Ophthalmic Genet. 2007 28 (3): 127-33

PMID: 17896310 · DOI:10.1080/13816810701503707

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.

MeSH Terms (11)

Animals Clinical Trials as Topic Disease Models, Animal Genetic Therapy Humans Macular Degeneration Optic Atrophy, Hereditary, Leber Photoreceptor Cells, Vertebrate Retinal Degeneration Retinal Neoplasms Retinoblastoma

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