Strategies for the treatment of repeat disease with RNA-binding compounds

Kazuhiko Nakatani

SANKEN Osaka University

Many repeat sequences in the human genome are associated with developing various neurodegenerative diseases.　Small molecules NA, NCD, and JM642, presented in this paper, showed remarkable activity in vivo.　NA shortened CAG repeats in the striatum of a mouse model of Huntington’s disease, NCD alleviated the degeneration of compound eyes in a Drosophila model of spinocerebellar degeneration, and JM642 alleviated splicing abnormalities in myotonic dystrophy type 1.　Although further studies are needed for all compounds, these compounds are expected to be important leads for developing drugs that can alleviate symptoms and even inhibit the onset of neurodegenerative diseases.

Address correspondence to Dr. Kazuhiko Nakatani, SANKEN Osaka University （8-1 Mihogaoka Ibaraki, 567-0047）