METHODS FOR TREATING NEURODEGENERATIVE DISEASES USING GENE THERAPY TO DELAY DISEASE ONSET AND PROGRESSION WHILE PROVIDING COGNITIVE PROTECTION

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专利权人:

UNIVERSITY OF IOWA RESEARCH FOUNDATION

发明人:

Beverly L. Davidson,Yong Hong Chen,Luis Tecedor

申请号：

US15769931

公开号：

US20180236105A1

申请日:

2016.10.24

申请国别(地区):

US

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.

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