Discovery of Clinically Approved Agents That Promote Suppression of Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations.

Venkateshwar MutyamMing DuXiaojiao XueKim M KeelingE Lucile WhiteJ Robert BostwickLynn RasmussenBo LiuMarina MazurJeong S HongEmily Falk LibbyFeng LiangHaibo ShangMartin MenseMark J SutoDavid M BedwellSteven M Rowe

Published in: American journal of respiratory and critical care medicine (2017)

Clinically approved drugs identified as potential readthrough agents, in combination with ivacaftor, may induce nonsense suppression to restore therapeutic levels of CFTR function. One or more agents may be suitable to advance to human testing.

Keyphrases

cystic fibrosis
pseudomonas aeruginosa
lung function
endothelial cells
small molecule
transcription factor
induced pluripotent stem cells
human health
climate change