Safety and Efficacy of Intravenous and Intrathecal Delivery of AAV9-Mediated ARSA in Minipigs

Safety and Efficacy of Intravenous and Intrathecal Delivery of AAV9-Mediated ARSA in Minipigs

Metachromatic leukodystrophy (MLD) is a hereditary neurodegenerative disease characterized by demyelination and motor and cognitive impairments due to deficiencies of the lysosomal enzyme arylsulfatase A (ARSA) or the saposin B activator protein (SapB). Current treatments are limited; however, gene...

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Bibliographic Details
Main Authors: Aysilu Mullagulova, Alisa Shaimardanova, Valeriya Solovyeva, Yana Mukhamedshina, Daria Chulpanova, Alexander Kostennikov, Shaza Issa, Albert Rizvanov
Format: Article
Language:English
Published: MDPI AG 2023-05-01
Series:International Journal of Molecular Sciences
Subjects:
adeno-associated virus
metachromatic leukodystrophy
arylsulfatase A
gene therapy
central nervous system
peripheral nervous system
Online Access:https://www.mdpi.com/1422-0067/24/11/9204

Internet

https://www.mdpi.com/1422-0067/24/11/9204

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