1. Lysosomal Storage Diseases: Heterogeneous Group of Disorders

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

Bioimpacts. 2013;3(4): 145-147. doi: 10.5681/bi.2013.029

PMCID: PMC3892733 PMID: 24455477 Scopus ID: 84893428618

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Original Research

2. 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.

Original Research

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

Mohammad A Rafi* , Paola Luzi, David A Wenger

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.

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