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...

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Dades bibliogràfiques
Publicat a:Ann Clin Transl Neurol
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: John Wiley and Sons Inc. 2020
Matèries:
Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7034505/
https://ncbi.nlm.nih.gov/pubmed/31967741
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.50975
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