ArticleMolecular therapy. Methods & clinical development2025
High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa.
Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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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
2 citing papers in PubMed.
- Highly potent MyoAAV4A vector reverses GSD III pathology in aged mice and enables long-term muscle disease correction in young adult mice.Molecular therapy. Advances · 2026Article
- WhenMolecular therapy. Methods & clinical development · 2025Article
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8 authors.
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Abstract
Glycogen storage disease type IIIa (GSD IIIa) affects multiple tissues, including liver, heart, and skeletal muscles. We recently reported that an adeno-associated virus serotype 9 vector expressing pullulanase, a bacterial glycogen debranching enzyme, driven by an immunotolerizing dual promoter (AAV9-Dual-Pull), effectively decreased pullulanase-induced cytotoxic T lymphocyte response and corrected disease abnormalities in all major affected tissues in GSD IIIa mice. To reduce effective vector dosages for transgene delivery to skeletal muscles, we packaged the AAV-Dual-Pull vector into two muscle-tropic MyoAAV capsids, MyoAAV4A and MyoAAV4E. Six weeks after administration of the same dose vector (1 × 10
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