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The synthetic aminoglycoside ELX-02 induces readthrough of G550X-CFTR producing superfunctional protein that can be further enhanced by CFTR modulators

Ten percent of cystic fibrosis (CF) patients carry a premature termination codon (PTC); no mutation-specific therapies exist for these individuals. ELX-02, a synthetic aminoglycoside, suppresses translation termination at PTCs (i.e., readthrough) by promoting the insertion of an amino acid at the PT...

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Detalles Bibliográficos
Autores principales:	Chen, Jianguo, Thrasher, Kari, Fu, Lianwu, Wang, Wei, Aghamohammadzadeh, Soheil, Wen, Hui, Tang, Liping, Keeling, Kim M., Falk Libby, Emily, Bedwell, David M., Rowe, Steven M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Physiological Society 2023
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10202470/ https://www.ncbi.nlm.nih.gov/pubmed/37014818 http://dx.doi.org/10.1152/ajplung.00038.2023

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