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....

Descripción completa

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

Internet

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

Ejemplares similares