Article
Discovery and SAR of 4-aminopyrrolidine-2-carboxylic acid correctors of CFTR for the treatment of cystic fibrosis.
Bioorganic & medicinal chemistry letters - 15 Sept 2022
Scanio Marc J C, Searle Xenia B, Liu Bo, Koenig John R, Altenbach Robert J, Gfesser Gregory A, Bogdan Andrew, Greszler Stephen, Zhao Gang, Singh Ashvani, Fan Yihong, Swensen Andrew M, Vortherms Timothy, Manelli Arlene, Balut Corina, Gao Wenqing, Yong Hong, Schrimpf Michael, Tse Chris, Kym Philip, Wang Xueqing
Abstract excerpt
Cystic fibrosis (CF) is an autosomal recessive disease resulting from mutations on both copies of the CFTR gene. Phenylalanine deletion at position 508 of the CFTR protein (F508del-CFTR) is the most frequent mutation in CF patients. Currently, the most effective treatments of CF use a dual or triple combination of CFTR correctors and potentiators. In triple therapy, two correctors (C1 and C2) and a potentiator...
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