NeurosciencebioRxivPreprint
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…·Nicolas Tricaud
This study shows that AAV2/9-mediated PMP22 silencing in rats prevents CMT1A disease and identifies skin biomarkers as potential outcome measures.
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