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THERAPIES GENIQUES POUR TROUBLES LYSOSOMIAUX
专利权人:
INC.;PREVAIL THERAPEUTICS
发明人:
ABELIOVICH, ASA,HECKMAN, LAURA,RHINN, HERVE
申请号:
CA3078464
公开号:
CA3078464A1
申请日:
2018.10.03
申请国别(地区):
CA
年份:
2019
代理人:
摘要:
The disclosure relates, in some aspects, to compositions and methods fortreatment of diseases associated with aberrantlysosomal function, for example Parkinson's disease (PD) and Gaucher disease.In some embodiments, the disclosure providesexpression constructs comprising a transgene encoding beta-Glucocerebrosidase(GBA) or a portion thereof alone or in combinationwith one or more PD-associated genes. In some embodiments, the disclosureprovides methods of Parkinson's disease byadministering such expression constructs to a subject in need thereof.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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