ASKEW, KIM,CHUNG, JOU-KU,DEROSA, FRANK,HEARTLEIN, MICHAEL,KARVE, SHRIRANG,MCCAULEY, THOMAS,SMITH, LIANNE,BARBIER, ANN J.
申请号：
CA3084061
公开号：
CA3084061A1
申请日:
2018.12.20
申请国别(地区):
CA
年份:
2019
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.
