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This study uses a new AAV-F capsid to treat a mouse model of pyruvate dehydrogenase complex deficiency, achieving improved survival and disease pathology with a dose ten times lower than the standard AAV9 approach. These findings highlight its strong potential for treating this severe neurodegenerative disorder
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Next Generation AAV-F capsid gene therapy rescues disease pathology in a model of Pyruvate Dehydrogenase Complex Deficiency
Molecular Therapy Family of Journals