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Ex Vivo Gene Therapy Approaches for the Treatment of Globoid Cell Leukodystrophy
2009
Globoid cell leukodystrophy (GLD) is a rare lysosomal storage disorder (LSD) due to the deficiency of the lysosomal enzyme Galactocerebrosidase (GALC). The enzymatic deficiency results in intracellular storage of undegraded metabolites in the nervous system, leading to progressive dysmyelination. We are testing the feasibility and efficacy of a gene therapy strategy based on hematopoietic stem/progenitor cells (HSPC) and lentiviral vectors (LV) in the murine model of GLD. Differently from what
doi:10.21954/ou.ro.0000f210
fatcat:dyntjy4iebekznxunzlpgsyctu