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THERAPIES GENIQUES POUR TROUBLES LYSOSOMAUX
专利权人:
INC.;PREVAIL THERAPEUTICS
发明人:
ABELIOVICH, ASA,HECKMAN, LAURA,RHINN, HERVE
申请号:
CA3078501
公开号:
CA3078501A1
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 and Gaucher disease. Insome embodiments, the disclosure provides expressionconstructs comprising a transgene encoding one or more inhibitory nucleicacids targeting SCNA or a portion thereof, THEM106B ora portion thereof, or any combination of the foregoing. In some embodiments,the disclosure provides methods of Parkinson's diseaseby administering 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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