ANTITHRIPSIN RNAI AGENTS (AAT) ALPHA-1, COMPOSITIONS INCLUDING AAT RNAI AGENTS, AND METHODS OF USE

摘要

these are rai agents to inhibit the expression of the alpha-1 antitrypsin gene (aat), compositions including rai agents of aat, and methods of use. there are also disclosed pharmaceutical compositions including one or more nanoparticles agents together with one or more excipients capable of delivering the nanoparticles agents to a liver cell in vivo. the delivery of aat renal agents to liver cells in vivo inhibits the expression of aat gene and treats diseases associated with aat deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis and fulminant liver failure.