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Article

HDAC6 is a therapeutic target in mutant GARS-induced Charcot-Marie-Tooth disease.

Brain : a journal of neurology - 1 Mar 2018

Benoy Veronick, Van Helleputte Lawrence, Prior Robert, d'Ydewalle Constantin, Haeck Wanda, Geens Natasja, Scheveneels Wendy, Schevenels Begga, Cader M Zameel, Talbot Kevin, Kozikowski Alan P, Vanden Berghe Pieter, Van Damme Philip, Robberecht Wim, Van Den Bosch Ludo

Abstract excerpt

Peripheral nerve axons require a well-organized axonal microtubule network for efficient transport to ensure the constant crosstalk between soma and synapse. Mutations in more than 80 different genes cause Charcot-Marie-Tooth disease, which is the most common inherited disorder affecting peripheral nerves. This genetic heterogeneity has hampered the development of therapeutics for Charcot-Marie-Tooth disease. The...

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Identifiers

PMID: 29415205
DOI: 10.1093/brain/awx375
PMCID: PMC5837793

Genes

HDAC6

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