Robust W1282X-CFTR rescue by a small molecule GSPT1 degrader
R. E. Lee, C. A. Lewis, L. He, S. C. Gallant, T. M. Mascenik, H. Dang, L. C. Morton, J. T. Minges, J. W. Theile, N. A. Castle, M. R. Knowles, A. J. Kimple, S. H. Randell
The paper develops Bmi-1/hTERT cell lines to model CFTR variants and shows that CC-90009 partially rescues W1282X-CFTR function while affecting sodium channel activity.
bioRxiv · Sep 28, 2021 · PreprintPathology