Abstract: 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.
November 16, 2018
Date of Patent:
March 30, 2021
THE SCRIPPS RESEARCH INSTITUTE, EXPANSION THERAPEUTICS, INC.
Matthew Disney, Timothy Allen Blizzard, Suzanne Rzuczek, John Ndungu, Joseph Vacca, Andy Jennings, Alexei Pushechnikov