ASKEW, Kim,CHUNG, Jou-ku,DEROSA, Frank,HEARTLEIN, Michael,KARVE, Shrirang,MCCAULEY, Thomas,SMITH, Lianne,BARBIER, Ann J.
申请号：
AU2018392716
公开号：
AU2018392716A1
申请日:
2018.12.20
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.
