Discovery and characterization of stereodefined PMO-gapmers targeting tau
Kunihiko Kanatsu, Yoshinori Takahashi, Tetsuya Sakaguchi, Dae‐Shik Kim, Miki Murota, Mingde Shan, Kazuki Fukami, Wataru Itano, Kenji Kikuta, Hikaru Yoshimura, Toshiki Kurokawa, Yuko Nagayama, Rena Ishikawa, Ryo Dairiki, Zhi Zhou, , Hyeong-Wook Choi
The paper develops a stereopure PMO-gapmer ASO to target tau, showing improved safety and activity by controlling phosphorus stereochemistry.
Molecular Therapy — Nucleic Acids · Nov 28, 2024Peer-reviewedNeuroscience