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Myosin7a Deficiency Results in Reduced Retinal Activity Which Is Improved by Gene Therapy

Mutations in MYO7A cause autosomal recessive Usher syndrome type IB (USH1B), one of the most frequent conditions that combine severe congenital hearing impairment and retinitis pigmentosa. A promising therapeutic strategy for retinitis pigmentosa is gene therapy, however its pre-clinical development...

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Bibliografiske detaljer
Main Authors: Colella, Pasqualina, Sommella, Andrea, Marrocco, Elena, Di Vicino, Umberto, Polishchuk, Elena, Garrido, Marina Garcia, Seeliger, Mathias W., Polishchuk, Roman, Auricchio, Alberto
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2013
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3753344/
https://ncbi.nlm.nih.gov/pubmed/23991031
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0072027
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