ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025
BIN1 gene replacement reverses BIN1-related centronuclear myopathy.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
1 citing paper in PubMed.
- Gene replacement therapy for centronuclear myopathy: A breakthrough in complex genetic muscle disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
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Authors and funding
6 authors.
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Abstract
Centronuclear myopathies (CNMs) are severe genetic disorders characterized by generalized muscle weakness associated with organelle mispositioning in myofibers. Most CNM cases are caused by mutations in proteins involved in membrane remodeling, including amphiphysin 2 (BIN1). There is no treatment, and the pathological mechanisms are not understood. Here, we aimed to cure the Bin1-CNM mouse model (Bin1
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Registered trials
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