Current development of CFTR potentiators in the last decade

Paola Barraja, Alessandra Montalbano, Maria Valeria Raimondi, Marilia Barreca, Virginia Spano', Arianna Venturini, Michele Genovese, Luis J.V. Galietta

Research output: Contribution to journalArticlepeer-review

18 Citations (Scopus)


Cystic fibrosis (CF) is a genetic disorder produced by the loss of function of CFTR, a main chloride channel involved in transepithelial salt and water transport. CFTR function can be rescued by small molecules called “potentiators” which increase gating activity of CFTR on epithelial surfaces. High throughput screening (HTS) assays allowed the identification of new chemical entities endowed with potentiator properties, further improved through medicinal chemistry optimization. In this review, the most relevant classes of CFTR potentiators developed in the last decade were explored, focusing on structure-activity relationships (SAR) of the different chemical entities, as a useful tool for the improvement of their pharmacological activity.
Original languageEnglish
Number of pages15
JournalEuropean Journal of Medicinal Chemistry
Publication statusPublished - 2020

All Science Journal Classification (ASJC) codes

  • Pharmacology
  • Drug Discovery
  • Organic Chemistry


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