Scientists from the Autonomous University of Barcelona have successfully used gene therapy to reverse motor dysfunction in mice afflicted with megalencephalic leukoencephalopathy with subcortical cysts (MLC). By delivering a healthy copy of the MLC1 gene through viral vectors, they restored normal brain function and mobility, even when treatment commenced after symptoms appeared. The study, published in Molecular Therapy, suggests that gene therapy could remain effective in patients who already show advanced disease — a hopeful advance for a condition that has few treatment options.
Source: MedicalXpress
Link: https://medicalxpress.com/news/2025-03-gene-therapy-reverses-symptoms-rare.html
Rare Neurological Disease Reversed in Animal Models via Gene Therapy
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