Metachromatic Leukodystrophy: Diagnosis, Modeling, and Treatment Approaches

Metachromatic Leukodystrophy: Diagnosis, Modeling, and Treatment Approaches

Metachromatic leukodystrophy is a lysosomal storage disease, which is characterized by damage of the myelin sheath that covers most of nerve fibers of the central and peripheral nervous systems. The disease occurs due to a deficiency of the lysosomal enzyme arylsulfatase A (ARSA) or its sphingolipid...

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Bibliographic Details
Main Authors: Alisa A. Shaimardanova, Daria S. Chulpanova, Valeriya V. Solovyeva, Aysilu I. Mullagulova, Kristina V. Kitaeva, Cinzia Allegrucci, Albert A. Rizvanov
Format: Article
Language:English
Published: Frontiers Media S.A. 2020-10-01
Series:Frontiers in Medicine
Subjects:
lysosomal storage diseases
metachromatic leukodystrophy
arylsulfatase A
sulfatide
replacement therapy
gene therapy
Online Access:https://www.frontiersin.org/articles/10.3389/fmed.2020.576221/full

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https://www.frontiersin.org/articles/10.3389/fmed.2020.576221/full

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