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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Publicat a:Bioimpacts
Autors principals: Rafi, Mohammad A, Luzi, Paola, Wenger, David A
Format: Artigo
Idioma:Inglês
Publicat: Tabriz University of Medical Sciences (TUOMS Publishing Group) 2021
Matèries:
Original Research
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8022232/
https://ncbi.nlm.nih.gov/pubmed/33842284
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.34172/bi.2021.21
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