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

Mohammad A. Rafi , Paola Luzi, David A. Wenger*

Bioimpacts. 2020;10(2): 105-115. doi: 10.34172/bi.2020.13

PMCID: PMC7186542 PMID: 32363154 Scopus ID: 85090729388

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Krabbe disease (KD) is an autosomal recessive lysosomal disorder that imposes defective myelination in the peripheral and central nervous systems. Twitcher combining bone marrow transplantation with AAVrh10-mGALC can have a great extension of life in the murine model of KD.

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