Delivery of large enzymes to the brain via transport across the blood-brain barrier (BBB) utilizing conjugates, or fusion proteins, which are composed of a therapeutic enzyme and a BBB targeting agent (molecular Trojan horse). The enzyme is missing in the brain, and does not cross the BBB. The molecular Trojan horse is a receptor-specific endogenous peptide, or peptidomimetic monoclonal antibody (MAb), that undergoes receptor-mediated transport across the BBB, thereby carrying into brain the attached enzyme.
DELIVERY OF ENZYMES TO THE BRAIN