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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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Detalles Bibliográficos
Autores principales: Colella, Pasqualina, Sommella, Andrea, Marrocco, Elena, Di Vicino, Umberto, Polishchuk, Elena, Garrido, Marina Garcia, Seeliger, Mathias W., Polishchuk, Roman, Auricchio, Alberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3753344/
https://www.ncbi.nlm.nih.gov/pubmed/23991031
http://dx.doi.org/10.1371/journal.pone.0072027