Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease
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 limited to hematopoietic...
Main Authors: | , , |
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Format: | Article |
Language: | English |
Published: |
Tabriz University of Medical Sciences
2020-03-01
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Series: | BioImpacts |
Subjects: | |
Online Access: | https://bi.tbzmed.ac.ir/PDF/bi-10-105.pdf |