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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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| Publicado no: | Int J Mol Sci |
|---|---|
| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
MDPI
2019
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| Assuntos: | |
| Acesso em linha: | 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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