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

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

Published: 2022-07-28

DOI: 10.1172/jci154571

Source: Full article


Abstract

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