WOODDELL, Christine, I. CI,LEWIS, David, L. DL,WAKEFIELD, Darren, H. DH,ALMEIDA, Lauren L,KANNER, Steven, B. SB,‧伍德爾,‧路易斯,‧維基菲爾德,‧埃爾米達,‧坎納
申请号：
HK17106007.9
公开号：
HK1232163A
申请日:
2017.06.16
申请国别(地区):
HK
年份:
2018
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.
