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 comprising one or more AAT RNAi agents together with one or more excipients capable of delivering the RNAi agent(s) to a liver cellin vivo. Delivery of the AAT RNAi agent(s) to liver cellsin vivoprovides for inhibition of AAT gene expression and treatment of diseases associated with AAT deficiency, such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.本發明描述用於抑制α-1抗胰蛋白酶(AAT)基因之表現的RNAi藥劑，包含AAT RNAi藥劑之組合物及使用方法。亦揭示醫藥組合物，其包括一或多種AAT RNAi藥劑與一或多種能夠活體內傳遞RNAi藥劑至肝細胞的賦形劑。AAT RNAi藥劑活體內傳遞至肝細胞提供對AAT基因表現之抑制以及治療與AAT缺乏相關的疾病，諸如慢性肝炎、肝硬化、肝細胞癌、轉胺酶升高、膽汁鬱積、纖維化及爆發性肝衰竭。
