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Original Research

1. Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease
Mohammad A. Rafi ORCID, Paola Luzi, David A. Wenger* ORCID
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.
Original Research

2. Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?

Mohammad A Rafi* ORCID, Paola Luzi, David A Wenger ORCID
BioImpacts. 2021;11(2): 135-146. doi: 10.34172/bi.2021.21
PMCID: PMC8022232     PMID: 33842284     Scopus ID: 85106877684    
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Our results demonstrate that, it may be possible to treat human KD patients with high dose AAVrh10 without blood stem cell transplantation which would eliminate the side effects of HSCT.
Mini Review

3. Krabbe disease: A personal perspective and hypothesis
Mohammad A. Rafi* ORCID
Bioimpacts. 2022;12(1): 3-7. doi: 10.34172/bi.2021.23931
PMCID: PMC8783082     PMID: 35087711     Scopus ID: 85127157009    
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Cutting viral dose in half or using BM cells from heterozygote mice in a combination of BMT and gene therapy treatments result in a similar reduction in lifespan of the treated mice.

As a peer-reviewed international open-access journal, BioImpacts publishes articles on basic and translational aspects of pharmaceutical and biomedical sciences. 
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