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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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Dettagli Bibliografici
Pubblicato in:Int J Mol Sci
Autori principali: Samanta, Ananya, Stingl, Katarina, Kohl, Susanne, Ries, Jessica, Linnert, Joshua, Nagel-Wolfrum, Kerstin
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6940777/
https://ncbi.nlm.nih.gov/pubmed/31842393
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20246274
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