CNS CNS delivery of therapeutic agents

摘要

The present invention provides an effective and less invasive approach for the direct delivery of therapeutic agents to the central nervous system (CNS). In certain embodiments, the present invention provides methods comprising intrathecally administering a composition comprising a replacement enzyme of a lysosomal enzyme to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced levels or activity of a lysosomal enzyme do.