The Trustees Of The University Of Pennsylvania;Christian Hinderer
发明人:
HINDERER, Christian,WILSON, James, M.
申请号：
AU2018335752
公开号：
AU2018335752A1
申请日:
2018.09.21
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hlDS gene and two or more immunosuppressants is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.
