Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency
Commun Med (Lond). 2025 Jan 27;5(1):29. doi: 10.1038/s43856-025-00734-9. ABSTRACT BACKGROUND: Multiple Sulfatase Deficiency (MSD) is a rare inherited lysosomal storage disorder characterized by loss of …