ArticleMolecular therapy. Advances2026
Highly potent MyoAAV4A vector reverses GSD III pathology in aged mice and enables long-term muscle disease correction in young adult mice.
Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
We recently reported a 6-week study in 3-month-old glycogen storage disorder (GSD) IIIa mice demonstrating that a myotropic AAV vector (MyoAAV4A-Dual-Pull) expressing a bacterial glycogen debranching enzyme (pullulanase) under the control of an immunotolerizing dual promoter achieved markedly higher transduction efficiency and superior glycogen clearance efficacy in cardiac and skeletal muscle compared with the AAV9-Dual-Pull vector. In the present study, we evaluated the efficacy of MyoAAV4A-Dual-Pull in aged GSD IIIa mice and assessed its therapeutic durability in young adult GSD IIIa mice. Intravenous injection of the MyoAAV4A-Dual-Pull (5 × 10
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