COLD SPRING HARBOR LABORATORY;Stoke Therapeutics, Inc.
发明人:
申请号:
EP16876606.1
公开号:
EP3390642A4
申请日:
2016.12.14
申请国别(地区):
EP
年份:
2019
代理人:
摘要:
Provided herein are methods and compositions for increasing the expression of PRPF3 or PRPF8, and for treating a subject in need thereof, e.g., a subject with deficient PRPF3 or PRPF8 protein expression or a subject having Retinitis Pigmentosa.