COLD SPRING HARBOR LABORATORY;Stoke Therapeutics, Inc.
发明人:
申请号：
EP16876621.0
公开号：
EP3389725A4
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 ATP1A2, CACNA1A, SETD5, SHANK3, NF2, DNMT1, TCF4, RAI1, PEX1, ARSA, EIF2B5, EIF2B1, EIF2B2, NPC1, ADAR, MFSD8, STXBP1, PRICKLE2, PRRT2, IDUA, or STX1B, and for treating a subject in need thereof, e.g., a subject with deficient ATP1A2, CACNA1A, SETD5, SHANK3, NF2, DNMT1, TCF4, RAI1, PEX1, ARSA, EIF2B5, EIF2B1, EIF2B2, NPC1, ADAR, MFSD8, STXBP1, PRICKLE2, PRRT2, IDUA, or STX1B protein expression.
