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Article

Effective therapeutic strategies in a preclinical mouse model of Charcot-Marie-Tooth disease.

Human molecular genetics - 30 Nov 2021

Nuevo-Tapioles Cristina, Santacatterina Fulvio, Sánchez-Garrido Brenda, de Arenas Cristina Núñez, Robledo-Bérgamo Adrián, Martínez-Valero Paula, Cantarero Lara, Pardo Beatriz, Hoenicka Janet, Murphy Michael P, Satrústegui Jorgina, Palau Francesc, Cuezva José M

Abstract excerpt

Charcot-Marie-Tooth (CMT) disease is a neuropathy that lacks effective therapy. CMT patients show degeneration of peripheral nerves, leading to muscle weakness and loss of proprioception. Loss of mitochondrial oxidative phosphorylation proteins and enzymes of the antioxidant response accompany degeneration of nerves in skin biopsies of CMT patients. Herein, we followed a drug-repurposing approach to find drugs in...

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Identifiers

PMID: 34274972
DOI: 10.1093/hmg/ddab207
PMCID: PMC8643506

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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