Neurocognitive testing in a murine model of mucopolysaccharidosis type IIIA
Mucopolysaccharidosis type IIIA (MPS IIIA) is an inherited metabolic disorder caused by a lysosomal enzyme deficiency resulting in heparan sulphate (HS) accumulation and manifests with a progressive neurodegenerative phenotype.A naturally occurring MPS IIIA mouse model is invaluable for preclinical evaluation of potential treatments but the ability