WOODDELL, CHRISTINE I.,LEWIS, DAVID L.,WAKEFIELD, DARREN H.,ALMEIDA, LAUREN,KANNER, STEVEN B.
申请号：
CA2951700
公开号：
CA2951700A1
申请日:
2015.06.16
申请国别(地区):
CA
年份:
2015
代理人:
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.
