AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy

Usher syndrome type III (USH3A) is an autosomal recessive disorder caused by mutations in clarin-1 (CLRN1) gene, leading to progressive retinal degeneration and sensorineural deafness. Efforts to develop therapies for preventing photoreceptor cell loss are hampered by the lack of a retinal phenotype...

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Detalles Bibliográficos
Autores principales: Dinculescu, Astra, Stupay, Rachel M., Deng, Wen-Tao, Dyka, Frank M., Min, Seok-Hong, Boye, Sanford L., Chiodo, Vince A., Abrahan, Carolina E., Zhu, Ping, Li, Qiuhong, Strettoi, Enrica, Novelli, Elena, Nagel-Wolfrum, Kerstin, Wolfrum, Uwe, Smith, W. Clay, Hauswirth, William W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2016
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4755610/
https://www.ncbi.nlm.nih.gov/pubmed/26881841
http://dx.doi.org/10.1371/journal.pone.0148874

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