AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

Gautier, Benoît; Hajjar, Hélène; Soares, Sylvia; Berthelot, Jade; Deck, Marie; Abbou, Scarlette; Campbell, G.; Ceprián, María; González, Sergio; Fovet, Claire-Maëlle; Schütza, Vlad; Jouvenel, Antoine; Rivat, Cyril; Zérah, Michel; François, Virginie; Le Guiner, Caroline; Aubourg, Patrick; Fledrich, Robert; Tricaud, Nicolas

doi:10.1038/s41467-021-22593-3

Public

AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

Published in Nature Communications • Apr 21, 2021

Authors:

Benoît Gautier

Hélène Hajjar

Sylvia Soares

Abstract

Charcot-Marie-Tooth disease 1 A (CMT1A) results from a duplication of the PMP22 gene in Schwann cells and a deficit of myelination in peripheral nerves. Patients with CMT1A have reduced nerve conduction velocity, muscle wasting, hand and foot deformations and foot drop walking. Here, we evaluate the...

Subject

Gene silencing

Sciatic nerve

Medicine

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