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...

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Bibliographic Details
Main Authors: Mohammad A. Rafi, Paola Luzi, David A. Wenger
Format: Article
Language:English
Published: Tabriz University of Medical Sciences 2020-03-01
Series:BioImpacts
Subjects:
Online Access:https://bi.tbzmed.ac.ir/PDF/bi-10-105.pdf