GNS-deficient mice show lysosomal storage CNS pathology, locomotor deficits, and shortened lifespan similar to humans with MPS IIID, and intracisternal administration of a vector encoding GNS (AAV9-GNS) reversed these deficits (Roca et al., 2017). This evidence concerns the gene GNS and mucopolysaccharidosis type 3D.