Long term AAV2/9-mediated silencing of PMP22 prevents CMT1A disease in rats and validates skin biomarkers as treatment outcome measure
Benoît Gautier, Hélène Hajjar, Sylvia Soares, Jade Berthelot, Marie Deck, Scarlette Abbou, Graham R. Campbell, Claire-Maëlle Fovet, Vlad Schütza, Antoine Jouvenel, Cyril Rivat, Michel Zérah, Virginie François Le Ravazet, Caroline Le Guiner, Patrick R. Aubourg, , Nicolas Tricaud
This study shows that AAV2/9-mediated PMP22 silencing in rats prevents CMT1A disease and identifies skin biomarkers as potential outcome measures.
bioRxiv · Jan 29, 2020 · PreprintNeuroscience