A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion

Premature termination codons (PTCs) prevent translation of a full-length protein and trigger nonsense-mediated mRNA decay (NMD). Nonsense suppression (also termed readthrough) therapy restores protein function by selectively suppressing translation termination at PTCs. Poor efficacy of current readt...

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Detalles Bibliográficos
Autores principales: Sharma, Jyoti, Du, Ming, Wong, Eric, Mutyam, Venkateshwar, Li, Yao, Chen, Jianguo, Wangen, Jamie, Thrasher, Kari, Fu, Lianwu, Peng, Ning, Tang, Liping, Liu, Kaimao, Mathew, Bini, Bostwick, Robert J., Augelli-Szafran, Corinne E., Bihler, Hermann, Liang, Feng, Mahiou, Jerome, Saltz, Josef, Rab, Andras, Hong, Jeong, Sorscher, Eric J., Mendenhall, Eric M., Coppola, Candice J., Keeling, Kim M., Green, Rachel, Mense, Martin, Suto, Mark J., Rowe, Steven M., Bedwell, David M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8285393/
https://www.ncbi.nlm.nih.gov/pubmed/34272367
http://dx.doi.org/10.1038/s41467-021-24575-x

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