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Gene therapies for lysosomal disorders
专利权人:
Inc.;Prevail Therapeutics
发明人:
Abeliovich, Asa,Heckman, Laura,Rhinn, Herve
申请号:
AU2020205228
公开号:
AU2020205228A1
申请日:
2020.07.14
申请国别(地区):
AU
年份:
2020
代理人:
摘要:
#$%^&*AU2020205228A120200730.pdf#####ABSTRACT The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and 5 Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof, Lysosomal Membrane Protein 2 (LIMP2), Prosaposin, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by 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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