David A. Wenger, Paola Luzi, Mohammad A. Rafi*
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Jaleh Barar  , Mohammad A. Rafi, Mohammad M. Pourseif, Yadollah Omidi*
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Masumeh Zamanlu, Morteza Eskandani*, Reza Mohammadian, Nazila Entekhabi, Mohammad Rafi, Mehdi Farhoudi*
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Mohammad A. Rafi*, Abass Alavi*
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Azam Safary , Mostafa Akbarzadeh Khiavi , Rahimeh Mousavi, Jaleh Barar , Mohammad A Rafi*
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Mohammad A. Rafi , Paola Luzi, David A. Wenger*
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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Roghaiyeh Ilghami , Hafez Mohammadhasanzadeh , Jaleh Barar , Mohammad A. Rafi*
Mohammad Rafi received his PhD in Animal Biology from the University of Montpellier, France, in 1970. Though he has worked on several lysosomal storage diseases, his main research interest is gene therapy of neurodegenerative disorders using animal models of globoid cell leukodystrophy (Krabbe disease). With successful AAVrh10-mediated treatment of murine and canine models, his research is moving towards the treatment trials of human patients.
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Mohammad A Rafi* , Paola Luzi, David A Wenger
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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Mohammad A. Rafi*
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.
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