RNA Interference Targeting Small Heat Shock Protein B8 Failed to Improve Distal Hereditary Motor Neuropathy in the Mouse Model.
Vendredy L; De Winter V; Van Lent J; Orije J; Authier TDS; Katona I; Asselbergh B; Adriaenssens E · 2025 · The journal of gene medicine
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Abstract (excerpt)
Background Missense mutations in the HSPB8 gene, encoding the small heat shock protein B8, cause distal hereditary motor neuropathy (dHMN) or an axonal form of Charcot-Marie-Tooth disease (CMT subtype 2L). Mice expressing mutant Hspb8…
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Metadata source: Europe PMC · DOI 10.1002/jgm.70013