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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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Main Authors: Colella, Pasqualina, Sommella, Andrea, Marrocco, Elena, Di Vicino, Umberto, Polishchuk, Elena, Garrido, Marina Garcia, Seeliger, Mathias W., Polishchuk, Roman, Auricchio, Alberto
格式: Artigo
語言:Inglês
出版: Public Library of Science 2013
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在線閱讀: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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