4.7 Article

Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knock-Out Mice

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Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

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Summary: Metachromatic leukodystrophy is a lysosomal storage disorder caused by a deficiency of arylsulfatase A, leading to myelin degeneration in the nervous system. Current treatments are ineffective for the most common late infantile form of the disease. Gene therapy has shown promise in alleviating symptoms in animal models and improving brain pathology in the short term, suggesting potential for use in rapidly progressing forms of the disease.

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