| abstract |
The invention describes an RNAi agent for inhibiting the expression of an α-1 antitrypsin (AAT) gene, a composition comprising the AAT RNAi agent, and a method of using the same. A pharmaceutical composition is also disclosed that includes one or more AAT RNAi agents and one or more excipients capable of delivering the RNAi agent to liver cells in vivo. AAT RNAi agents are delivered in vivo to liver cells to provide inhibition of AAT gene expression and to treat diseases associated with AAT deficiency, such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, elevated transaminases, cholestasis, fibrosis, and explosiveness Liver failure. |