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Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease

[Image: see text] Introduction: Krabbe disease (KD) is an autosomal recessive lysosomal disorder caused by mutations in the galactocerebrosidase (GALC) gene. This results in defective myelination in the peripheral and central nervous systems due to low GALC activity. Treatment at this time is limite...

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Detalhes bibliográficos
Publicado no:Bioimpacts
Main Authors: Rafi, Mohammad A., Luzi, Paola, Wenger, David A.
Formato: Artigo
Idioma:Inglês
Publicado em: Tabriz University of Medical Sciences 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7186542/
https://ncbi.nlm.nih.gov/pubmed/32363154
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.34172/bi.2020.13
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