Abstract
In the past years we worked extensively on the development of HSC gene therapy for two fatal demyelinating lysosomal storage disorders, Metachromatic (MLD) and Globoid Cell (GLD) leukodystrophies. We recently demonstrated that transplantation of lentiviral vector-transduced Hematopoietic Stem Cells (HSC) prevented and corrected functional and pathological manifestations of MLD in the mouse model (Biffi A. et al., JCI 2004; Biffi A. et al, submitted for publication). Moreover, we proved the critical role of Arylsulfatase A (ARSA) over-expression to attain therapeutic efficacy.
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