Article

Evaluating Intravenous and Intrathecal Administration of AAV Encoding ARSA Gene Therapy Approaches for Metachromatic Leukodystrophy in Minipigs

2023-04-19

Abstract excerpt

Metachromatic leukodystrophy (MLD) is a hereditary neurodegenerative disease characterized by demye-lination and motor and cognitive impairment due to the deficiency of the lysosomal enzyme arylsulfatase A (ARSA) or the saposin B activator protein (SapB). Current treatments are limited; however, gene therapy using adeno-associated virus (AAV) vectors for ARSA delivery has shown promising results. The main challeng...

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Literature Corpus work: b42fa84d-2402-522d-a5a8-31bda0f9ff69
DOI: 10.20944/preprints202304.0539.v1

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