Article

Treatment of Adult Metachromatic Leukodystrophy Model Mice Using Intrathecal Administration of Type 9 AAV Vector Encoding Arylsulfatase A

2021-07-12

Abstract excerpt

Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by an arylsulfatase A (ASA) deficiency and characterized by severe neurological symptoms resulting from demyelination within the central and peripheral nervous systems. We investigated the feasibility and efficacy of intrathecal administration of a type 9 adeno-associated viral vector encoding ASA (AAV9/ASA) for the treatment of 6-week-old ML...

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Literature Corpus work: 4a8dc631-2ec6-54e4-8e48-4f9ef4c8323b
DOI: 10.21203/rs.3.rs-687914/v1

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