Ontology highlight
ABSTRACT:
SUBMITTER: Mills AD
PROVIDER: S-EPMC3165007 | biostudies-other | 2010 Jan
REPOSITORIES: biostudies-other
Mills Aaron D AD Yoo Choong C Butler Jeffrey D JD Yang Baoxue B Verkman A S AS Kurth Mark J MJ
Bioorganic & medicinal chemistry letters 20091113 1
A developing therapy of cystic fibrosis caused by the DeltaF508 mutation in CFTR employs correction of defective CFTR chloride channel gating by a 'potentiator' and of defective CFTR protein folding by a 'corrector'. Based on SAR data for phenylglycine-type potentiators and bithiazole correctors, we designed a hybrid molecule incorporating an enzymatic hydrolysable linker to deliver the potentiator (PG01) fragment 2 and the corrector (Corr-4a) fragment 13. The hybrid molecule 14 contained PG01-O ...[more]