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...

Täydet tiedot

Bibliografiset tiedot

Päätekijät:	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
Aineistotyyppi:	Artigo
Kieli:	Inglês
Julkaistu:	American Society for Clinical Investigation 2022-09-01
Sarja:	The Journal of Clinical Investigation
Aiheet:	Pulmonology Therapeutics
Linkit:	https://doi.org/10.1172/JCI154571
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