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

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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Bibliographic Details
Main Authors: Rhianna E. Lee, Catherine A. Lewis, Lihua He, Emily C. Bulik-Sullivan, Samuel C. Gallant, Teresa M. Mascenik, Hong Dang, Deborah M. Cholon, Martina Gentzsch, Lisa C. Morton, John T. Minges, Jonathan W. Theile, Neil A. Castle, Michael R. Knowles, Adam J. Kimple, Scott H. Randell
Format: Article
Language:English
Published: American Society for Clinical Investigation 2022-09-01
Series:The Journal of Clinical Investigation
Subjects:
Pulmonology
Therapeutics
Online Access:https://doi.org/10.1172/JCI154571

Internet

https://doi.org/10.1172/JCI154571

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