Research Group for Nucleic Acid-Targeted Small Molecule Drug Discovery / ISIR / Osaka University

Development and Evaluation of Small Molecules Binding to Repeat Nucleic Acids

We are participating as a co-researcher in the research project "Development of Fundamental Therapies through Shortening Abnormally Expanded Repeats in Triplet Repeat Diseases," led by Professor Masayuki Nakamori, Faculty of Medicine, Yamaguchi University, under the AMED program "Practical Research Project for Rare/Intractable Diseases." Professor Nakatani has successfully developed small molecules that bind to CAG repeats (Huntington's disease), CTG (CUG) repeats (Myotonic Dystrophy Type 1), CGG re..