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METHODS AND COMPOSITIONS FOR THE TREATMENT OF ALS
专利权人:
UNIVERSITY OF CINCINNATI
发明人:
Alexander John MacLennan
申请号:
US16088570
公开号:
US20200297868A1
申请日:
2017.03.31
申请国别(地区):
US
年份:
2020
代理人:
摘要:
A method for treating a subject suffering from a motor neuron degenerative disorder is provided herein, the method including: administering to the subject one or more modified adeno-associated virus (AAV) gene delivery vectors packaging a recombinant AAV (rAVV)-based genome, wherein each AAV vector is engineered to include a cDNA insert selected from a ciliary neutrophic factor receptor alpha (CNTFRa) cDNA insert, a cardiotrophin-like cytokine factor 1 (CLC) cDNA insert, and a cytokine receptor-like factor 1 (CLF) cDNA insert. Also provided are pharmaceutical compositions including one or more modified AAV gene delivery vectors, each AAV vector packaging a rAAV genome engineered to include (i) a cDNA insert selected from CNTFRa, CLC, and CLF; and (ii) a promoter; and a pharmaceutically acceptable excipient.
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中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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