Design of an orally bioavailable small molecule that modulates the microtubule-associated protein tau's pre-mRNA splicing
Peiyuan Zhang, Amirhossein Taghavi, Masahito Abe, Haruo Aikawa, Yoshihiro Akahori, Jonathan L. Chen, Yuquan Tong, Jared T. Baisden, Michael D. Cameron, Jessica L. Childs‐Disney, Matthew David Disney
This paper designs an orally bioavailable small molecule that targets RNA to reduce 4R tau protein abundance and alleviate disease phenotypes in a mouse model.
ACS Chemical Biology · Jun 23, 2025Peer-reviewedBiochemistry