WOODDELL CHRISTINE I,LEWIS DAVID L,WAKEFIELD DARREN H,ALMEIDA LAUREN,KANNER STEVEN B
申请号：
IN201627042045
公开号：
IN201627042045A
申请日:
2016.12.08
申请国别(地区):
IN
年份:
2017
代理人:
摘要：
in vivoin vivoThe 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 . Delivery of the AAT RNAi trigger to liver cells provides for inhibition of AAT gene expression and treatment of alpha 1 antitrypsin deficiency and associated diseases.
