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Brainstem development requires galactosylceramidase and is critical for pathogenesis in a model of Krabbe disease

Krabbe disease (KD) is caused by a deficiency of galactosylceramidase (GALC), which induces demyelination and neurodegeneration due to accumulation of cytotoxic psychosine. Hematopoietic stem cell transplantation (HSCT) improves clinical outcomes in KD patients only if delivered pre-symptomatically....

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Detalles Bibliográficos
Autores principales:	Weinstock, Nadav I., Kreher, Conlan, Favret, Jacob, Nguyen, Duc, Bongarzone, Ernesto R., Wrabetz, Lawrence, Laura Feltri, M., Shin, Daesung
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7584660/ https://www.ncbi.nlm.nih.gov/pubmed/33097716 http://dx.doi.org/10.1038/s41467-020-19179-w

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