CFTR structures bound to ETI components predict rare mutation response to modulator combinations
Noemie Stanleigh, Michal Gur, Michal Shteinberg, Aryeh M. Weiss, Naama Sebbag-Sznajder, Deborah Duran, Myriam Grunewald, Liron Birimberg‐Schwartz, Ronen Bar‐Yoseph, Jeffrey Matthijn Beekman, Eitan Kerem, Michael A. Wilschanski, Batsheva Kerem
The paper predicts and validates responses to CFTR modulator combinations in patients with rare mutations using structural data and functional assays.
medRxiv · Oct 12, 2024 · PreprintGenetic & Genomic Medicine