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- W2029563036 abstract "MLD is inborn lipidosis caused by the deficiency of the lysosomal enzymes arylsulfatase A (ARSA), which results in storage of undergraded sulfatides in the CNS and PNS, leading to progressive demyelination. Bone marrow transplant provides a clinically feasible method for permanently replacing deficient lysosomal enzyme activity. Using Lentiviral Vectors (LV), we efficiently transduced hematopoietic stem cells (HSC) ex vivo and evaluated the potential of their progeny to target therapeutic genes to the CNS and PNS of transplanted mice, and correct MLD phenotype in the mouse model. We proved extensive repopulation of CNS microglia and PNS endoneurial macrophages by transgene-expressing cells. By transplanting HSC transduced with the ARSA gene, we reconstituted enzyme activity in the hematopoietic system of MLD mice at supranormal levels and prevented major disease manifestations: the complete protection from the development of the motor conduction impairment and of the neuropathological abnormalities (prevention of sulfatide accumulation and demyelination in CNS and PNS) typical of the disease. Remarkably, ex vivo gene therapy had a significantly higher therapeutic impact than wild-type HSC transplantation on all examined parameters, highlighting the crucial role of enzyme over-expression and indicating the likely occurrence of in vivo cross-correction." @default.
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- W2029563036 date "2004-06-01" @default.
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- W2029563036 title "Correction of metachromatic leukodystrophies (MLD) in the mouse model by transplantation of genetically modified hematopoietic stem cells" @default.
- W2029563036 doi "https://doi.org/10.1111/j.1085-9489.2004.009209af.x" @default.
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