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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...
Enregistré dans:
| Publié dans: | Int J Mol Sci |
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| Auteurs principaux: | , , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
MDPI
2019
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| Sujets: | |
| Accès en ligne: | 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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