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 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...

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Detalles Bibliográficos
Autores principales: Gautier, Benoit, Hajjar, Helene, Soares, Sylvia, Berthelot, Jade, Deck, Marie, Abbou, Scarlette, Campbell, Graham, Ceprian, Maria, Gonzalez, Sergio, Fovet, Claire-Maëlle, Schütza, Vlad, Jouvenel, Antoine, Rivat, Cyril, Zerah, Michel, François, Virginie, Le Guiner, Caroline, Aubourg, Patrick, Fledrich, Robert, Tricaud, Nicolas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8060274/
https://www.ncbi.nlm.nih.gov/pubmed/33883545
http://dx.doi.org/10.1038/s41467-021-22593-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8060274/
https://www.ncbi.nlm.nih.gov/pubmed/33883545
http://dx.doi.org/10.1038/s41467-021-22593-3

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