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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Publicado no:Nat Commun
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2021
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8285393/
https://ncbi.nlm.nih.gov/pubmed/34272367
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-021-24575-x
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