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Funding Awarded

1 award

  • Ex vivo autologous stem cell gene therapy for Multiple Sulfatase Deficiency

    Multiple Sulfatase Deficiency (MSD) is an inherited lysosomal storage disorder (LSD) that predominately involves the brain, bones, and skin. The disease is due to mutations in SUMF1, a gene that provides the instructions for making an important enzyme called formylglycine generating enzyme (FGE). The FGE enzyme activates all the...