6533b7d4fe1ef96bd1263431
RESEARCH PRODUCT
Current development of CFTR potentiators in the last decade
Maria Valeria RaimondiPaola BarrajaLuis J. V. GaliettaMichele GenoveseVirginia SpanòAlessandra MontalbanoArianna VenturiniMarilia Barrecasubject
congenital hereditary and neonatal diseases and abnormalitiesHigh-throughput screeningGlycineComputational biologyQuinolonesVX-770Aminophenols01 natural sciencesCystic fibrosisCystic fibrosisSmall Molecule LibrariesStructure-Activity Relationship03 medical and health sciencesDrug DiscoverymedicineHumansCFTR potentiatorCFTRLoss function030304 developmental biologyPharmacology0303 health sciencesWater transportbiology010405 organic chemistryChemistryOrganic ChemistryCFTR potentiatorsBiological activityGeneral MedicineTriazolesPotentiatormedicine.diseaseCystic fibrosis transmembrane conductance regulator0104 chemical sciencesCystic fibrosiMutationChloride channelbiology.proteinCystic fibrosis transmembrane conductance regulatordescription
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.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2020-01-01 | European Journal of Medicinal Chemistry |