LI, ZHEN,ZHU, RUI,PEI, TAO,NICHOLAS, ANTHONY,BUSH, ERIK W.
申请号：
CA3061752
公开号：
CA3061752A1
申请日:
2018.07.05
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an alpha-ENaC (SCNN1A) gene. The alpha-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an alpha-ENaC gene. Pharmaceutical compositions that include one or more alpha-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described alpha-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of alpha-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects.
