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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...
Autores principales: | , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2016
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Materias: | |
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 |