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Metachromatic leukodystrophy and transplantation: remyelination, no cross‐correction
OBJECTIVE: 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 a...
Autores principales: | Wolf, Nicole I., Breur, Marjolein, Plug, Bonnie, Beerepoot, Shanice, Westerveld, Aimee S. R., van Rappard, Diane F., de Vries, Sharon I., Kole, Maarten H. P., Vanderver, Adeline, van der Knaap, Marjo S., Lindemans, Caroline A., van Hasselt, Peter M., Boelens, Jaap J., Matzner, Ulrich, Gieselmann, Volkmar, Bugiani, Marianna |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7034505/ https://www.ncbi.nlm.nih.gov/pubmed/31967741 http://dx.doi.org/10.1002/acn3.50975 |
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