We have recently described the efficacy of AAV2/8-mediated liver gene transfer in animal models of mucopolysaccharidosis VI (MPS VI), an LSD characterized by glycosaminoglycan (GAG) storage in various tissues including liver (Fig. 2A) due to arylsulfatase B (ARSB) deficiency [14], [15], [34]. The gene discussed is ARSB; the disease is mucopolysaccharidosis type 6.