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

Vanda S. Lopes | Zendy; David S. Williams | Zendy

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

Author(s) -

Vanda S. Lopes,

David S. Williams

Publication year - 2015

Publication title -

cold spring harbor perspectives in medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 3.853

H-Index - 105

eISSN - 2472-5412

pISSN - 2157-1422

DOI - 10.1101/cshperspect.a017319

Subject(s) - usher syndrome , retinal degeneration , blindness , genetic enhancement , gene , medicine , retinitis pigmentosa , genetics , biology , optometry

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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