MPS VI mice received at postnatal day 30 (p30) a single i.v. administration of either 2 × 1011 or 6 × 1011 gc/kg of AAV2/8.TBG.hARSB, which encodes human ARSB (hARSB) under the control of the liver-specific thyroxine-binding globulin (TBG) promoter, and/or monthly i.v. injections of 1 mg/kg rhARSB (Naglazyme, BioMarin Europe, London, UK), which is the dose currently used in MPS VI patients management.6,7,49,50,51,52. This evidence concerns the gene SERPINA7 and mucopolysaccharidosis type 6.