In Vitro Modulator Responsiveness of 655 CFTR Variants Found in People With CF
H. Bihler, A. Sivachenko, L. Millen, P. Bhatt, A. Thakerar Patel, J. Chin, V. Bailey, I. Musisi, A. LaPan, N. E. Allaire, J. Conte, N. R. Simon, A. S. Magaret, K. S. Raraigh, G. R. Cutting, , M. Mense
This study evaluates the responsiveness of 655 CFTR variants to the modulator therapy ELX/TEZ/IVA, identifying variants that show improved chloride transport function.
bioRxiv · Jul 7, 2023 · PreprintPharmacology & Toxicology