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AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome

CF Viscomi, M Zeviani

OAI: oai:www.repository.cam.ac.uk:1810/265944 • DOI: 10.17863/CAM.10762

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Abstract

mitochondrial disease Leigh syndrome AAV gene therapy Ndufs4 Mouse models experimental therapy