Breakthrough therapies: Cystic fibrosis (CF) potentiators and correctors.

Academic Article

Abstract

  • Cystic Fibrosis is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene resulting in abnormal protein function. Recent advances of targeted molecular therapies and high throughput screening have resulted in multiple drug therapies that target many important mutations in the CFTR protein. In this review, we provide the latest results and current progress of CFTR modulators for the treatment of cystic fibrosis, focusing on potentiators of CFTR channel gating and Phe508del processing correctors for the Phe508del CFTR mutation. Special emphasis is placed on the molecular basis underlying these new therapies and emerging results from the latest clinical trials. The future directions for augmenting the rescue of Phe508del with CFTR modulators are also emphasized.
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    Keywords

  • CFTR modulators, CFTR molecular defect, cystic fibrosis, lung disease, novel therapies, Aminophenols, Aminopyridines, Animals, Benzodioxoles, Clinical Trials as Topic, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Drug Design, Homozygote, Humans, Mice, Molecular Targeted Therapy, Mutation, Protein Folding, Quinolones
  • Digital Object Identifier (doi)

    Authorlist

  • Solomon GM; Marshall SG; Ramsey BW; Rowe SM
  • Start Page

  • S3
  • End Page

  • S13
  • Volume

  • 50 Suppl 40