Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?

[Image: see text] Introduction: Krabbe disease (KD) is an autosomal recessive disorder caused by mutations in the galactocerebrosidase (GALC) gene resulting in neuro-inflammation and defective myelination in the central and peripheral nervous systems. Most infantile patients present with clinical fe...

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Detalles Bibliográficos
Autores principales: Rafi, Mohammad A, Luzi, Paola, Wenger, David A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Tabriz University of Medical Sciences (TUOMS Publishing Group) 2021
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8022232/
https://www.ncbi.nlm.nih.gov/pubmed/33842284
http://dx.doi.org/10.34172/bi.2021.21

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