CAGリピート伸長を標的とした非環型人工核酸SNA修飾siRNAの開発です。

K. Maeda, T. Hirunagi, K. Sahashi, Y. Kamiya, M. Iida, K. Sakakibara, K. Onodera, M. Ohyama, Y. Okada, H. Okano, H. Asanuma, M. Katsuno, An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment. Mol. Ther. Nucleic Acids 37, 102802 (2026). https://doi.org/10.1016/j.omtn.2025.102802