LEVIN, ARTHUR A.,GEALL, ANDREW JOHN,COCHRAN, MICHAEL CARAMIAN,HUANG, HANHUA,DOPPALAPUDI, VENKATA RAMANA,BURKE, ROB,DARIMONT, BEATRICE DIANA,SHI, YUNYU,JOHNS, RACHEL
申请号：
CA3075425
公开号：
CA3075425A1
申请日:
2018.09.21
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
Disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion.
