Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy

Blood-brain barrier disruption marks the onset of cerebral adrenoleukodystrophy (CALD), a devastating cerebral demyelinating disease caused by loss of ABCD1 gene function. The underlying mechanism are not well understood, but evidence suggests that microvascular dysfunction is involved. We analyzed...

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Detalles Bibliográficos
Autores principales: Lauer, Arne, Speroni, Samantha L., Choi, Myoung, Da, Xiao, Duncan, Christine, McCarthy, Siobhan, Krishnan, Vijai, Lusk, Cole A., Rohde, David, Hansen, Mikkel Bo, Kalpathy-Cramer, Jayashree, Loes, Daniel J., Caruso, Paul A., Williams, David A., Mouridsen, Kim, Emblem, Kyrre E., Eichler, Florian S., Musolino, Patricia L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10076264/
https://www.ncbi.nlm.nih.gov/pubmed/37019892
http://dx.doi.org/10.1038/s41467-023-37262-w

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10076264/
https://www.ncbi.nlm.nih.gov/pubmed/37019892
http://dx.doi.org/10.1038/s41467-023-37262-w

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