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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...

Täydet tiedot

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Bibliografiset tiedot
Julkaisussa:Int J Mol Sci
Päätekijät: Samanta, Ananya, Stingl, Katarina, Kohl, Susanne, Ries, Jessica, Linnert, Joshua, Nagel-Wolfrum, Kerstin
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: MDPI 2019
Aiheet:
Linkit: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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