Article

Muscle-specific DNM2 overexpression improves Charcot-Marie-Tooth disease in vivo and reveals a narrow therapeutic window in skeletal muscle

2025-11-27

Abstract excerpt

Charcot-Marie-Tooth disease (CMT) caused by dominant loss-of-function mutations in DNM2 , encoding the GTPase dynamin-2, impairs motor and sensory function. However, the respective contributions of muscle and nerve pathology, and the therapeutic potential of increasing DNM2 expression, remain unresolved. We evaluated tissue-targeted and systemic approaches to increase DNM2 in a mouse model carrying the common K56...

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Literature Corpus work: 4b60e108-aa88-5576-8b56-34519c470d29
DOI: 10.1101/2025.11.25.690395

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