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

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

Charcot-Marie-Tooth disease 1 A (CMT1A) results from PMP22 gene duplication and is characterized by peripheral nerve myelination deficits. Here, the authors prevent the development of pathological features in a rat model of CMT1A through the local delivery of AAV2/9 expressing shRNAs against PMP22....

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Bibliographic Details
Main Authors: Benoit Gautier, Helene Hajjar, Sylvia Soares, Jade Berthelot, Marie Deck, Scarlette Abbou, Graham Campbell, Maria Ceprian, Sergio Gonzalez, Claire-Maëlle Fovet, Vlad Schütza, Antoine Jouvenel, Cyril Rivat, Michel Zerah, Virginie François, Caroline Le Guiner, Patrick Aubourg, Robert Fledrich, Nicolas Tricaud
Format: Article
Language:English
Published: Nature Publishing Group 2021-04-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-021-22593-3

Internet

https://doi.org/10.1038/s41467-021-22593-3

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