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...

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Detalles Bibliográficos
Autores principales: Wang, Dan, Xue, Xiaojiao, Gunn, Gwen, Du, Ming, Siddiqui, Amna, Weetall, Marla, Keeling, Kim M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2022
Materias:
Original Article
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

Internet

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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