Small-molecule drugs for cystic fibrosis: Where are we now?

doi:10.1016/j.pupt.2021.102098

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Article

Small-molecule drugs for cystic fibrosis: Where are we now?

Pulmonary pharmacology & therapeutics - 1 Feb 2022

Laselva Onofrio, Guerra Lorenzo, Castellani Stefano, Favia Maria, Di Gioia Sante, Conese Massimo

Abstract excerpt

The cystic fibrosis (CF) lung disease is due to the lack/dysfunction of the CF Transmembrane Conductance Regulator (CFTR), a chloride channel expressed by epithelial cells as the main regulator of ion and fluid homeostasis. More than 2000 genetic variation in the CFTR gene are known, among which those with identified pathomechanism have been divided into six mutation classes. A major advancement in the...

Topics

Aminophenols
Chloride Channels
Cystic Fibrosis
Cystic Fibrosis Transmembrane Conductance Regulator
Humans
Mutation
Prospective Studies

Source

Open in PubMed

Identifiers

PMID: 34793977
DOI: 10.1016/j.pupt.2021.102098

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