864. Characterization of Transgenic Mice Over_ Expressing Arylsulfatase A: Implications for Gene Therapy and Sulfatases Activation Mechanisms

Abstract

We have recently shown that in order to attain therapeutic efficacy by HSC gene therapy for Metachromatic Leukodystrophy (MLD), Arylsulfatase A (ARSA) over_expression in HSC and their progeny is required. The recent identification of Sulfatase Modifying Factor 1 (SUMF1) as a common activator of sulfatases and a rate_limiting factor in the biological activation of these enzymes, raises concerns for possible adverse effects of enzyme over_expression within certain cell type (Cosma M.P. et al., Cell 2003).