Arthur A. LEVIN,Andrew John GEALL,Venkata Ramana DOPPALAPUDI,Michael Caramian COCHRAN,Hanhua HUANG,Rob BURKE
申请号：
US16128450
公开号：
US20180369400A1
申请日:
2018.09.11
申请国别(地区):
US
年份:
2018
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.
