DIMER COMPOUNDS, AND USE IN BINDING TOXIC REPEATS OF RNA
申请人:EXPANSION THERAPEUTICS, INC.
公开号:US20200207744A1
公开(公告)日:2020-07-02
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.
Bis-benzimidazole compounds and methods of using the same
申请人:THE SCRIPPS RESEARCH INSTITUTE
公开号:US10961202B2
公开(公告)日:2021-03-30
Provided herein are compounds and methods for modulating abnormal repeat expansions of gene sequences. More particularly, provided are 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.