Article

AAV-mediated ARSA replacement for the treatment of Metachromatic Leukodystrophy

2025-03-13

Abstract excerpt

<h4>ABSTRACT</h4> Metachromatic leukodystrophy (MLD) is an autosomal recessive neurodegenerative disorder caused by mutations in the arylsulfatase A (ARSA) gene, resulting in lower sulfatase activity and the toxic accumulation of sulfatides in the central and peripheral nervous system. Children account for 70% of cases and become progressively disabled with death occurring within 10 years of disease onset. Gene t...

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Literature Corpus work: 8c93bd1e-19c3-51c6-bfef-904a5ac65c88
DOI: 10.1101/2025.03.12.642609

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