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Journal of the American Chemical Society|May 5, 2020
Design, Optimization, and Study of Small Molecules That Target Tau Pre-mRNA and Affect SplicingJonathan L Chen, Peiyuan Zhang, Masahito Abe, et al.Journal of the American Chemical Society|October 6, 2025
Covalent Probes Reveal Small-Molecule Binding Pockets in Structured RNA and Enable Bioactive Compound DesignSandra Kovachka, Jielei Wang, Amirhossein Taghavi, et al.Proceedings of the National Academy of Sciences of the United States of America|November 21, 2022
A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(G4C2)exp in c9ALS/FTD via the nuclear RNA exosomeJessica A Bush, Samantha M Meyer, Rita Fuerst, et al.Nature|May 24, 2023
Programming inactive RNA-binding small molecules into bioactive degradersYuquan Tong, Yeongju Lee, Xiaohui Liu, et al.Science Translational Medicine|October 27, 2021
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS modelsJessica A Bush, Haruo Aikawa, Rita Fuerst, et al.Pageof 9