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...

Popoln opis

Bibliografske podrobnosti

Principais autores:	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:	Artigo
Jezik:	Inglês
Izdano:	American Society for Clinical Investigation 2022-09-01
Serija:	The Journal of Clinical Investigation
Teme:	Pulmonology Therapeutics
Online dostop:	https://doi.org/10.1172/JCI154571
Oznake:	Označite Brez oznak, prvi označite!