Small-molecule eRF3a degraders rescue CFTR nonsense mutations by promoting premature termination codon readthrough

The vast majority of people with cystic fibrosis (CF) are now eligible for CF transmembrane regulator (CFTR) modulator therapy. The remaining individuals with CF harbor premature termination codons (PTCs) or rare CFTR variants with limited treatment options. Although the clinical modulator response...

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Detalles Bibliográficos
Autores principales: Lee, Rhianna E., Lewis, Catherine A., He, Lihua, Bulik-Sullivan, Emily C., Gallant, Samuel C., Mascenik, Teresa M., Dang, Hong, Cholon, Deborah M., Gentzsch, Martina, Morton, Lisa C., Minges, John T., Theile, Jonathan W., Castle, Neil A., Knowles, Michael R., Kimple, Adam J., Randell, Scott H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9479597/
https://www.ncbi.nlm.nih.gov/pubmed/35900863
http://dx.doi.org/10.1172/JCI154571

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