ARSB and mucopolysaccharidosis type 6: In addition, gene therapy at the dose of 2 × 1012 gc/kg was at least as effective as weekly infusions of recombinant human ARSB (rhARSB) in a mouse model of MPS VI, suggesting that it may be preferable to ERT as it potentially requires a single i.v. administration.15 If this translates to humans, gene therapy could significantly improve the quality of life of MPS VI patients.