Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy.

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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Bibliographic Details
Main Authors: Pasqualina Colella, Andrea Sommella, Elena Marrocco, Umberto Di Vicino, Elena Polishchuk, Marina Garcia Garrido, Mathias W Seeliger, Roman Polishchuk, Alberto Auricchio
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2013-01-01
Series:PLoS ONE
Online Access:https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0072027&type=printable
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https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0072027&type=printable

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