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Ataluren suppresses a premature termination codon in an MPS I-H mouse
ABSTARCT: Suppressing translation termination at premature termination codons (PTCs), termed readthrough, is a potential therapy for genetic diseases caused by nonsense mutations. Ataluren is a compound that has shown promise for clinical use as a readthrough agent. However, some reports suggest tha...
Autores principales: | Wang, Dan, Xue, Xiaojiao, Gunn, Gwen, Du, Ming, Siddiqui, Amna, Weetall, Marla, Keeling, Kim M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Berlin Heidelberg
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9329424/ https://www.ncbi.nlm.nih.gov/pubmed/35857082 http://dx.doi.org/10.1007/s00109-022-02232-0 |
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