GENE TRANSFER COMPOSITIONS, METHODS AND USES FOR TREATING NEURODEGENERATIVE DISEASES

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专利权人:

The Children's Hospital of Philadelphia

发明人:

Beverly L. DAVIDSON,Yong Hong CHEN,Luis TECEDOR

申请号：

US16344298

公开号：

US20190269797A1

申请日:

2017.11.03

申请国别(地区):

US

年份:

2019

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.

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