Cold Spring Harbor Laboratory;Stoke Therapeutics, Inc.
发明人:
Isabel ARNAREZ,Huw M. NASH
申请号：
US16062286
公开号：
US20180369275A1
申请日:
2016.12.14
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
