Mohammad Morad Farajollahi, Sepideh Hamzehlou, Ahmad Mehdipour, Ali Samadikuchaksaraei*
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Jaleh Barar  , Yadollah Omidi*
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Jaleh Barar , Yadollah Omidi*
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Jaleh Barar , Yadollah Omidi*
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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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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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Mojtaba Ghobadi , Mohammad Foad Heidari, Arezoo Farhadi, Ali Shakerimoghaddam, Mahdi Ghorbani, Zahra Hami, Naeim Ehtesham, Javad Behroozi*
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