ALFA-1-ANTITRIPSIN (AAT) RNAs, COMPOSITIONS CONTAINING AAT RNAs, AND WAYS OF THEIR APPLICATION

摘要

RNAi agents for inhibiting alpha-1-antitrypsin gene expression (AAT), compositions comprising RNAi AAT agents, and methods for their use are described. Also described are pharmaceutical compositions comprising one or more RNAi AAT agents together with one or more excipients capable of delivering an RNAi agent (s) to an in vivo liver cell. The delivery of RNAi AAT agent (s) to liver cells in vivo inhibits AAT gene expression and treats diseases associated with AAT deficiency, such as chronic hepatitis, cirrhosis, hepatic cell carcinoma, transaminitis, cholestasis, fibrosis, and fulminant liver failure.