Genetic & Genomic MedicinemedRxivPreprint
CFTR structures bound to ETI components predict rare mutation response to modulator combinations
Noemie Stanleigh, Michal Gur, Michal Shteinberg…·Batsheva Kerem
The paper predicts and validates responses to CFTR modulator combinations in patients with rare mutations using structural data and functional assays.
DOI 10.1101/2024.10.12.24314979