Michael Heartlein,Frank DeRosa,Jonathan Cherry,Paula Lewis,Shrirang Karve,Zarna Patel,Darshan Parekh,Susan Wood,Chi-Sung Chiu,Caroline J. Woo
申请号：
US16012138
公开号：
US20180369413A1
申请日:
2018.06.19
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present invention provides methods and compositions of treating Friedreichs ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.
