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Gene therapy rescues brain edema and motor function in a mouse model of megalencephalic leukoencephalopathy with subcortical cysts.
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Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles.
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Megalencephalic leukoencephalopathy with subcortical cysts: a variant update and review of the literature.
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Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain.
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Cerebellar Astrocyte Transduction as Gene Therapy for Megalencephalic Leukoencephalopathy.
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Seizures and disturbed brain potassium dynamics in the leukodystrophy megalencephalic leukoencephalopathy with subcortical cysts.
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Disrupting MLC1 and GlialCAM and ClC-2 interactions in leukodystrophy entails glial chloride channel dysfunction.
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