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Shayam Ramachandran on Muck Rack

Shayam Ramachandran

As seen in: BioRxiv
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Articles

AAV-mediated ARSA replacement for the treatment of Metachromatic Leukodystrophy

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By Jie Bu, Shayam Ramachandran, Jeffery Ardinger, MiAngela Ramos
| BioRxiv
Abstract 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.
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