COLD SPRING HARBOR LABORATORY;STOKE THERAPEUTICS, INC.
发明人:
AZNAREZ, ISABEL,NASH, HUW M.,KRAINER, ADRIAN
申请号：
CA3005245
公开号：
CA3005245A1
申请日:
2016.12.13
申请国别(地区):
CA
年份:
2017
代理人:
摘要：
Provided herein are methods and compositions for increasing the expression of JAGl, and for treating a subject in need thereof, e.g., a subject with deficient JAGl protein expression or a subject having Alagille syndrome (ALGS).
