Padmanabh Chivukula,Luigi Warren,Kiyoshi Tachikawa,Joseph E. Payne
申请号：
US15806096
公开号：
US20180051262A1
申请日:
2017.11.07
申请国别(地区):
US
年份:
2018
代理人:
摘要：
This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.
