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In metachromatic leukodystrophy, a lysosomal storage disorder due to decreased arylsulfatase A activity, hematopoietic stem cell transplantation may stop brain demyelination and allow remyelination, thereby halting white matter degeneration. This… Click to show full abstract
In metachromatic leukodystrophy, a lysosomal storage disorder due to decreased arylsulfatase A activity, hematopoietic stem cell transplantation may stop brain demyelination and allow remyelination, thereby halting white matter degeneration. This is the first study to define the effects and therapeutic mechanisms of hematopoietic stem cell transplantation on brain tissue of transplanted metachromatic leukodystrophy patients.
Keywords:
metachromatic leukodystrophy;
transplantation remyelination;
leukodystrophy transplantation;
leukodystrophy
Journal Title: Annals of Clinical and Translational Neurology
Year Published: 2020
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Journal Title: Annals of Clinical and Translational Neurology
Year Published: 2020
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!