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Article

Proteostasis Regulators in Cystic Fibrosis: Current Development and Future Perspectives.

Journal of medicinal chemistry - 14 Apr 2022

Brusa Irene, Sondo Elvira, Falchi Federico, Pedemonte Nicoletta, Roberti Marinella, Cavalli Andrea

Abstract excerpt

In cystic fibrosis (CF), the deletion of phenylalanine 508 (F508del) in the CF transmembrane conductance regulator (CFTR) leads to misfolding and premature degradation of the mutant protein. These defects can be targeted with pharmacological agents named potentiators and correctors. During the past years, several efforts have been devoted to develop and approve new effective molecules. However, their clinical use...

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Identifiers

PMID: 35377645
DOI: 10.1021/acs.jmedchem.1c01897
PMCID: PMC9014417

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