TECEDOR, Luis;CHEN, Yong Hong;DAVIDSON, Beverly;UNIVERSITY OF IOWA RESEARCH FOUNDATION
发明人:
DAVIDSON, Beverly,CHEN, Yong Hong,TECEDOR, Luis
申请号:
SG11201803218P
公开号:
SG11201803218PA
申请日:
2016.10.24
申请国别(地区):
SG
年份:
2018
代理人:
摘要:
The present disclosure provides methods of treating a lysosomal storage disorder in a mammal which method comprises administering AAV particles encoding a polypeptide directly to the central nervous system of the mammal in conjunction with administering at least two immunosuppressive agents. AAV particles may be delivered by direct injection into the brain, spinal cord, cerebral spinal fluid or a portion thereof.