COLD SPRING HARBOR LABORATORY;STOKE THERAPEUTICS, INC.
发明人:
AZNAREZ, ISABEL,NASH, HUW M.,KRAINER, ADRIAN
申请号：
CA3005246
公开号：
CA3005246A1
申请日:
2016.12.14
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 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.
