Abstract
Spontaneous canine models exist for several inherited retinal dystrophies. This review will summarize the models and indicate where they have been used in translational gene therapy trials. The RPE65 gene therapy trials to treat childhood blindness are a good example of how studies in dogs have contributed to therapy development. Outcomes in human clinical trials are compared and contrasted with the result of the preclinical dog trials.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Animals
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Blindness / genetics
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Blindness / therapy*
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Color Vision Defects / genetics
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Color Vision Defects / therapy
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Disease Models, Animal*
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Dogs
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Genetic Therapy*
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Humans
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Leber Congenital Amaurosis / genetics
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Leber Congenital Amaurosis / therapy
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Macular Degeneration / genetics
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Macular Degeneration / therapy
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Retinal Dystrophies / genetics
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Retinal Dystrophies / therapy*
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cis-trans-Isomerases / genetics
Substances
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retinoid isomerohydrolase
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cis-trans-Isomerases