Calias, Pericles,Pan, Jing,Powell, Jan,Charnas, Lawrence,McCauley, Thomas,Shahrokh, Zahra,Wright, Teresa Leah,Pfeifer, Richard
申请号：
AU2011270668
公开号：
AU2011270668A1
申请日:
2011.06.25
申请国别(地区):
AU
年份:
2013
代理人:
摘要：
The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.
