[HTML][HTML] Correctors and Potentiators Rescue Function of the Truncated W1282X-Cystic Fibrosis Transmembrane Regulator (CFTR) Translation Product*♦
PM Haggie, PW Phuan, JA Tan, H Xu… - Journal of Biological …, 2017 - ASBMB
W1282X is the fifth most common cystic fibrosis transmembrane regulator (CFTR) mutation
that causes cystic fibrosis. Here, we investigated the utility of a small molecule
corrector/potentiator strategy, as used for ΔF508-CFTR, to produce functional rescue of the
truncated translation product of the W1282X mutation, CFTR 1281, without the need for read-
through. In transfected cell systems, certain potentiators and correctors, including VX-809
and VX-770, increased CFTR 1281 activity. To identify novel correctors and potentiators with …
that causes cystic fibrosis. Here, we investigated the utility of a small molecule
corrector/potentiator strategy, as used for ΔF508-CFTR, to produce functional rescue of the
truncated translation product of the W1282X mutation, CFTR 1281, without the need for read-
through. In transfected cell systems, certain potentiators and correctors, including VX-809
and VX-770, increased CFTR 1281 activity. To identify novel correctors and potentiators with …