首页>
外国专利>
Compositions and Methods for Inhibiting Gene Expression of Alpha-1 AntiTrypsin
Compositions and Methods for Inhibiting Gene Expression of Alpha-1 AntiTrypsin
展开▼
机译:抑制α-1抗胰蛋白酶基因表达的组合物和方法
展开▼
页面导航
摘要
著录项
相似文献
摘要
The invention relates to a RNA interference triggers for inhibiting the expression of an AAT gene through the mechanism of RNA interference. The invention also relates to a pharmaceutical composition comprising the AAT RNAi trigger together with an excipient capable of improving delivery of the RNAi trigger to a liver cell in vivo. Delivery of the AAT RNAi trigger to liver cells in vivo provides for inhibition of AAT gene expression and treatment of alpha 1-antitrypsin deficiency and associated diseases.
展开▼