Wird geladen...

Intrathecal Administration of AAV/GALC Vectors in 10-11 Day old Twitcher Mice Improves Survival and is Enhanced by Bone Marrow Transplant

Globoid cell Leukodystrophy (GLD), or Krabbe disease, is an autosomal recessive neurodegenerative disease caused by the deficiency of the lysosomal enzyme Galactocerebrosidase (GALC). Hematopoietic stem cell transplantation (HSCT) provides modest benefit in pre-symptomatic patients but well short of...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:J Neurosci Res
Hauptverfasser: Karumuthil-Melethil, Subha, Marshall, Michael S., Heindel, Clifford, Jakubauskas, Benas, Bongarzone, Ernesto, Gray, Steven J.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2016
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5028109/
https://ncbi.nlm.nih.gov/pubmed/27638599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jnr.23882
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!