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The Journal of Clinical Investigation|May 17, 2022
A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A miceMarina Stavrou, Alexia Kagiava, Sarah G Choudury, et al.Brain : a Journal of Neurology|February 11, 2022
NCAM1 and GDF15 are biomarkers of Charcot-Marie-Tooth disease in patients and miceMatthew J Jennings, Alexia Kagiava, Leen Vendredy, et al.Molecular Therapy. Nucleic Acids|April 8, 2026
Safety, efficacy, and distal nerve Schwann cell biodistribution in mice and NHPs to support translation of AAV9 RNAi therapy for CMT1AMarina Stavrou, Lindsay M Wallace, Merlin P Thangaraj, et al.Pageof 2