An overview on chemical structures as ΔF508-CFTR correctors

Alessandra Montalbano, Paola Barraja, Virginia Spano', Anna Carbone, Paolo Scudieri, Luis J.V. Galietta

Research output: Contribution to journalArticle

3 Citations (Scopus)

Abstract

Deletion of phenylalanine at position 508 (F508del) in the CFTR protein, is the most common mutation causing cystic fibrosis (CF). F508del causes misfolding and rapid degradation of CFTR protein a defect that can be targeted with pharmacological agents termed "correctors". Correctors belong to various chemical classes but are generally small molecules based on nitrogen sulfur or oxygen heterocycles. The mechanism of action of correctors is generally unknown but there is experimental evidence that some of them can directly act on mutant CFTR improving folding and stability. Here we overview the characteristics of the various F508del correctors described so far to obtain indications on key chemical structures and modifications that are required for mutant protein rescue.
Original languageEnglish
Pages (from-to)430-448
Number of pages19
JournalEuropean Journal of Medicinal Chemistry
Volume180
Publication statusPublished - 2019

All Science Journal Classification (ASJC) codes

  • Pharmacology
  • Drug Discovery
  • Organic Chemistry

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