Article
CFTR modulator theratyping: Current status, gaps and future directions.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2019
Clancy John Paul, Cotton Calvin U, Donaldson Scott H, Solomon George M, VanDevanter Donald R, Boyle Michael P, Gentzsch Martina, Nick Jerry A, Illek Beate, Wallenburg John C, Sorscher Eric J, Amaral Margarida D, Beekman Jeffrey M, Naren Anjaparavanda P, Bridges Robert J, Thomas Philip J, Cutting Garry, Rowe Steven, Durmowicz Anthony G, Mense Martin, Boeck Kris D, Skach William, Penland Christopher, Joseloff Elizabeth, Bihler Hermann, Mahoney John, Borowitz Drucy, Tuggle Katherine L
Abstract excerpt
BACKGROUND: New drugs that improve the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein with discreet disease-causing variants have been successfully developed for cystic fibrosis (CF) patients. Preclinical model systems have played a critical role in this process, and have the potential to inform researchers and CF healthcare providers regarding the nature of defects in rare...
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