CNS DELIVERY OF THERAPEUTIC AGENTS

摘要

PROBLEM TO BE SOLVED: To provide a CNS delivery of therapeutic agents.SOLUTION: The invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the invention provides methods comprising a step of intrathecally administering a composition containing the anaplerotic enzyme of a lysosomal enzyme to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of the lysosomal enzyme. Many lysosomal storage disorders affect the nervous system, and thus we demonstrate unique challenges in treating these diseases with traditional therapies.SELECTED DRAWING: None