Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder characterized by accumulation of sulfatides in both glial cells and neurons. MLD results from an inherited deficiency of arylsulfatase A (ARSA) and myelin degeneration in the central and peripheral nervous systems. Currently, no effe...

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Bibliographic Details
Main Authors: Emilie Audouard, Valentin Oger, Béatrix Meha, Nathalie Cartier, Caroline Sevin, Françoise Piguet
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-05-01
Series:Frontiers in Molecular Neuroscience
Subjects:
metachromatic leukodystrophy
aav
gene therapy
intravenous injection (i.v.)
sulfatide accumulation
lysosomal storage disease
Online Access:https://www.frontiersin.org/articles/10.3389/fnmol.2021.677895/full

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https://www.frontiersin.org/articles/10.3389/fnmol.2021.677895/full

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