DAVIDSON, BEVERLY L.,CHEN, YONG HONG,TECEDOR, LUIS
申请号：
CA3041548
公开号：
CA3041548A1
申请日:
2017.11.03
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
Provided are methods of treating a lysosomal storage disorder in a mammal which method includes administering AAV particles encoding a polypeptide to the central nervous system of the mammal. AAV particles may be delivered by direct injection into the brain, spinal cord, cerebral spinal fluid or a portion thereof for expression.
