NeuroscienceMolecular Therapy — Nucleic AcidsPeer-reviewed
Discovery and characterization of stereodefined PMO-gapmers targeting tau
Kunihiko Kanatsu, Yoshinori Takahashi, Tetsuya Sakaguchi…·Hyeong-Wook Choi
The paper develops a stereopure PMO-gapmer ASO to target tau, showing improved safety and activity by controlling phosphorus stereochemistry.