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Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
专利权人:
Arrowhead Pharmaceuticals; Inc.
发明人:
Zhen Li,Rui Zhu,Christine I. Wooddell,Tao Pei
申请号:
US15867107
公开号:
US10450565B2
申请日:
2018.01.10
申请国别(地区):
US
年份:
2019
代理人:
摘要:
RNAi agents for inhibiting the expression of the alpha-1 antitrypsin (AAT) gene, compositions including AAT RNAi agents, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more AAT RNAi agents together with one or more excipients capable of delivering the RNAi agent(s) to a liver cell in vivo. Delivery of the AAT RNAi agent(s) to liver cells in vivo inhibits AAT gene expression and treats diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.
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http://www.ckcest.cn/home/

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