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Customized Antisense Oligonucleotide-Based Therapy for Neurofilament-Associated Charcot-Marie-Tooth Disease

Jessica Medina,Adriana Rebelo,Matt C. Danzi, Elizabeth H. Jacobs,Isaac R. L. Xu, Kathleen P. Ahrens, Sitong Chen, Jacquelyn Raposo,Christopher Yanick,Stephan Zuchner,Mario A. Saporta

BRAIN（2024）

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Key words
antisense oligonucleotide,induced pluripotent stem cell,Charcot-Marie-Tooth disease,therapeutic,axonal degeneration
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