An overview on chemical structures as ΔF508-CFTR correctors

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

Risultato della ricerca: Articlepeer review

3 Citazioni (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.
Lingua originaleEnglish
pagine (da-a)430-448
Numero di pagine19
RivistaEuropean Journal of Medicinal Chemistry
Volume180
Stato di pubblicazionePublished - 2019

All Science Journal Classification (ASJC) codes

  • Pharmacology
  • Drug Discovery
  • Organic Chemistry

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