Early pathological signs in young dysf -/- mice are improved by halofuginone.

Neuromuscular Disorders(2020)

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摘要
•Dysferlinopathies are a group of late-onset muscular dystrophies.•Impaired fusion of primary dysf −/− myoblasts from young mice.•Elevated fibrotic symptoms are found in dysf−/− mice at 4 weeks of age.•Early halofuginone treatment rescues dysf−/− mouse muscle histopathology.•Early halofuginone treatment improves myoblast fusion in dysf−/− mice.
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关键词
Dysferlinopathy,Differentiation,Halofuginone,Fibrosis,Fusion,Hypertrophy
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