In Vivo Brain Delivery of BBB-Enabled Iduronate 2-Sulfatase in Rats

Delivery of Enzyme Replacement Therapy Across the Blood-Brain Barrier for Hunter Syndrome Background Hunter syndrome (Mucopolysaccharidosis II, MPS II) is a rare inherited metabolic disorder caused by a deficiency of the iduronate-2-sulfatase (IDS) enzyme. This enzyme deficiency leads to the accumulation of glycosaminoglycans (such as heparan sulfa...