DAVIDSON, Beverly L.,CHEN, Yong Hong,TECEDOR, Luis
申请号：
EP17867272
公开号：
EP3534892A4
申请日:
2017.11.03
申请国别(地区):
EP
年份:
2020
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.
