Given the urgent need to identify therapies that can either cure or significantly alleviate the symptoms of this severely debilitating disease, as well as other MPS types that accumulate pathogenic HS fragments and exhibit a similar CNS phenotype, we have recently developed a novel therapeutic approach utilizing an HS binding protein: the recombinant hepatocyte growth factor/scatter factor (HGF/SF) natural spliced variant, NK1 [16, 23–25]. Here, HGF is linked to mucopolysaccharidosis.