Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulators
Author:
Funder
NIH
Cystic Fibrosis Foundation
Cystic Fibrosis Foundation Therapeutics
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://insight.jci.org/articles/view/121159/files/pdf
Reference79 articles.
1. Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complementary DNA
2. The ABC protein turned chloride channel whose failure causes cystic fibrosis
3. Cystic fibrosis genetics: from molecular understanding to clinical application;Cutting;Nat Rev Genet,2015
4. Transformative therapies for rare CFTR missense alleles
5. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770
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