EXPANSION THERAPEUTICS, INC.;THE SCRIPPS RESEARCH INSTITUTE
发明人:
Timothy Allen Blizzard,Matthew Disney
申请号：
US16728248
公开号：
US20200207744A1
申请日:
2019.12.27
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Provided herein are compounds and methods for modulating abnormal repeat expansions of gene sequences. More particularly, provided are dimeric inhibitors of RNA and the uses of such inhibitors in regulating nucleotide repeat expansions, e.g., to treat Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2 (DM2), Fuchs dystrophy, Huntington Disease, Amyotrophic Lateral Sclerosis, or Frontotemporal Dementia.
