73289-90-4Relevant articles and documents
DIMER COMPOUNDS, AND USE IN BINDING TOXIC REPEATS OF RNA
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, (2020/07/16)
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.