The Board of Trustees of the Leland Stanford Junior University
发明人:
Mark A. Kay,Anton McCaffrey
申请号：
US16723744
公开号：
US20200246370A1
申请日:
2019.12.20
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Methods and compositions are provided for modulating, e.g., reducing, coding sequence expression in mammals. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is administered to a non-embryonic mammal, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.
