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Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations

The identification of genetic defects that underlie inherited retinal diseases (IRDs) paves the way for the development of therapeutic strategies. Nonsense mutations caused approximately 12% of all IRD cases, resulting in a premature termination codon (PTC). Therefore, an approach that targets nonse...

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Detalles Bibliográficos
Autores principales:	Samanta, Ananya, Stingl, Katarina, Kohl, Susanne, Ries, Jessica, Linnert, Joshua, Nagel-Wolfrum, Kerstin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6940777/ https://www.ncbi.nlm.nih.gov/pubmed/31842393 http://dx.doi.org/10.3390/ijms20246274

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