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Article

Pharmacological chaperone action in humanized mouse models of MC4R-linked obesity.

JCI insight - 22 Feb 2021

René Patricia, Lanfray Damien, Richard Denis, Bouvier Michel

Abstract excerpt

MC4R mutations represent the largest monogenic cause of obesity, resulting mainly from receptor misfolding and intracellular retention by the cellular quality control system. The present study aimed at determining whether pharmacological chaperones (PCs) that restore folding and plasma membrane trafficking by stabilizing near native protein conformation may represent valid therapeutic avenues for the treatment of...

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Identifiers

PMID: 33434184
DOI: 10.1172/jci.insight.132778
PMCID: PMC7934941

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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