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...

Brain Endothelial Permeability, Transport, and Flow Assessed Over 10 Orders of Magnitude Using the In Situ Brain Perfusion Technique

Assessment of Brain Endothelial Permeability, Transport, and Flow Background Introduction The blood-brain barrier (BBB) is a critical barrier that protects the brain from harmful substances while also serving as a key obstacle for drug delivery to the brain. The permeability and transport properties of the BBB directly influence the distribution of...