Emerging Cystic Fibrosis Transmembrane Conductance Regulator Modulators as New Drugs for Cystic Fibrosis: A Portrait of in Vitro Pharmacology and Clinical Translation
Drishti P Ghelani, Elena K Schneider-Futschik
ACS PHARMACOLOGY & TRANSLATIONAL SCIENCE | AMER CHEMICAL SOC | Published : 2020
Pharmacological correction of the defective ion channel with cystic fibrosis transmembrane conductance regulator (CFTR) has become an attractive approach to therapy directed at the root cause of the life-limiting disease cystic fibrosis (CF). CFTR defects range from absence, misfolding, and resulting degradation to functional defects of the CFTR protein. The discovery and development of the CFTR potentiator ivacaftor was a major break-through in CF therapy and has triggered an enormous incentive for seeking effective modulators such as lumacaftor, tezacaftor or elexacaftor for all patients with CF. A number of emerging CFTR modulators are currently in the development pipeline, and rescue lev..View full abstract
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E.K.S.-F. is supported by a research grant from The University of Melbourne and the Peter Phelan Research Award from The Thoracic Society of Australia and New Zealand. E.K.S.-F. is also supported by the Australian National Health and Medical Research Council (NHMRC) as a Biomedical Research Fellow. The content is solely the responsibility of the authors and does not necessarily represent the official views of the Australian National Health and Medical Research Council.