Network Biology Identifies Novel Regulators of CFTR Trafficking and Membrane Stability.

ABSTRACT: In cystic fibrosis, the most common disease-causing mutation is F508del, which causes not only intracellular retention and degradation of CFTR, but also defective channel gating and decreased membrane stability of the small amount that reaches the plasma membrane (PM). Thus, pharmacological correction of mutant CFTR requires targeting of multiple cellular defects in order to achieve clinical benefit. Although small-molecule compounds have been identified and commercialized that can correct its folding or gating, an efficient retention of F508del CFTR at the PM has not yet been explored pharmacologically despite being recognized as a crucial factor for improving functional rescue of chloride transport. In ongoing efforts to determine the CFTR interactome at the PM, we used three complementa

SUBMITTER: Loureiro CA 

PROVIDER: S-EPMC6559121 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature