ASO-enhancement of TARDBP exitron splicing mitigates TDP-43 proteinopathies
Takuma Yamagishi, Shingo Koide, Genri Toyama, Aya Washida, Yumi Yamada, Ryutaro Hanyu, Ekaterina Nadbitova, Yuka Mitsuhashi Koike, Takuya Konno, Tomohiko Ishihara, Taisuke Kato, Osamu Onodera, Akihiro Sugai
This paper shows that enhancing TARDBP exitron splicing reduces TDP-43 aggregation and neurodegeneration in disease models using ASOs.
bioRxiv · Jul 22, 2024 · PreprintNeuroscience