Gene therapy reverses the symptoms of a rare neurological disease in animal models
Megaloencephalic leukoencephalopathy with subcortical cysts (MLC) is a rare childhood disease that mainly affects the white matter of the brain. Among other symptoms, it is characterized by macrocephaly, inability to coordinate motor movements, and epilepsy. More than 75% of diagnosed cases are due to mutations in the MLC1 gene, which encodes a protein located in […]
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