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Korea University College of Medicine

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World’s first mouse model precisely replicating the MT-ND4 gene mutation (G11778A) responsible for Leber’s Hereditary Optic Neuropathy (LHON). Using AAV-based TALED mitochondrial gene-editing technology, the research team successfully corrected the mutation in both the mouse model and patient-derived cells, experimentally demonstrating the therapeutic potential of gene editing.

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KU Medicine

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