52336-92-2Relevant academic research and scientific papers
SMALL MOLECULES THAT TARGET THE RNA THAT CAUSES ALS
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, (2022/04/03)
Disclosed herein are compounds that selectively bind an expanded transcribed repeat r(G4C2)exp, prevent sequestration of RNA-binding proteins, and inhibit translation of repeat associated non-ATG (RAN) translation responsible for generation of toxic dipeptide repeats underlying diseases such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The compounds and their pharmaceutical compositions are useful in treating a disease or condition characterized by an expanded G4C2 repeat RNA (r(G4C2)exp), such as ALS and FTD.
