Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A

Usher syndrome is a deaf-blindness disorder. One of the subtypes, Usher 1B, is caused by loss of function of the gene encoding the unconventional myosin, MYO7A. A variety of different viral-based delivery approaches have been tested for retinal gene therapy to prevent the blindness of Usher 1B, and...

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Bibliographic Details
Published inCold Spring Harbor perspectives in medicine Vol. 5; no. 6; p. a017319
Main Authors Lopes, Vanda S, Williams, David S
Format Journal Article
LanguageEnglish
Published United States Cold Spring Harbor Laboratory Press 01.06.2015
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Summary:Usher syndrome is a deaf-blindness disorder. One of the subtypes, Usher 1B, is caused by loss of function of the gene encoding the unconventional myosin, MYO7A. A variety of different viral-based delivery approaches have been tested for retinal gene therapy to prevent the blindness of Usher 1B, and a clinical trial based on one of these approaches has begun. This review evaluates the different approaches.
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ISSN:2157-1422
2472-5412
DOI:10.1101/cshperspect.a017319