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Mitochondrial gene therapy shows potential application in Leber’s hereditary optic neuropathy (LHON)

Research published in the US journal Proceedings of the National Academy of Sciences (PNAS) has shown that gene delivery to mitochondria is capable of suppressing visual loss and optic atrophy in an experimental model of Leber’s hereditary optic neuropathy (LHON). The results may be expected to have important implications not only for LHON but also for a broad range of human disorders brought about through mitochondrial gene defects.