CFTR Modulators: From Mechanism to Targeted Therapeutics

Han I. Yeh, Katy J. Sutcliffe, David N. Sheppard, Tzyh Chang Hwang*

*Corresponding author for this work

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

2 Scopus citations

Abstract

People with cystic fibrosis (CF) suffer from a multi-organ disorder caused by loss-of-function variants in the gene encoding the epithelial anion channel cystic fibrosis transmembrane conductance regulator (CFTR). Tremendous progress has been made in both basic and clinical sciences over the past three decades since the identification of the CFTR gene. Over 90% of people with CF now have access to therapies targeting dysfunctional CFTR. This success was made possible by numerous studies in the field that incrementally paved the way for the development of small molecules known as CFTR modulators. The advent of CFTR modulators transformed this life-threatening illness into a treatable disease by directly binding to the CFTR protein and correcting defects induced by pathogenic variants. In this chapter, we trace the trajectory of structural and functional studies that brought CF therapies from bench to bedside, with an emphasis on mechanistic understanding of CFTR modulators.

Original languageEnglish
Title of host publicationHandbook of Experimental Pharmacology
PublisherSpringer Science and Business Media Deutschland GmbH
Pages219-247
Number of pages29
DOIs
StatePublished - 2024

Publication series

NameHandbook of Experimental Pharmacology
Volume283
ISSN (Print)0171-2004
ISSN (Electronic)1865-0325

Keywords

  • ABC transporters
  • Chloride channels
  • Cystic fibrosis
  • Pharmacology

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