scAAV9/SUMF1 shows safety and efficacy as gene replacement therapy for the treatment of multiple sulfatase deficiency
Feb. 13, 2025
Multiple sulfatase deficiency is an autosomal recessive lysosomal storage disorder caused by homozygous or compound heterozygous loss-of-function mutations in the SUMF1 gene, which encodes formylglycine generating enzyme (FGE), which catalyzes the post-translational modification of all known 17 sulfatases.