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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Bibliografiske detaljer
Udgivet i:Bioimpacts
Main Authors: Rafi, Mohammad A., Luzi, Paola, Wenger, David A.
Format: Artigo
Sprog:Inglês
Udgivet: Tabriz University of Medical Sciences 2020
Fag:
Original Research
Online adgang: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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