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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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Vydáno v:Cold Spring Harb Perspect Med
Hlavní autoři: Lopes, Vanda S., Williams, David S.
Médium: Artigo
Jazyk:Inglês
Vydáno: Cold Spring Harbor Laboratory Press 2015
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4448706/
https://ncbi.nlm.nih.gov/pubmed/25605753
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/cshperspect.a017319
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