Discovery and characterization of stereodefined PMO-gapmers targeting tau

Discovery and characterization of stereodefined PMO-gapmers targeting tau

Antisense oligonucleotides (ASOs) are an important class of therapeutics to treat genetic diseases, and expansion of this modality to neurodegenerative disorders has been an active area of research. To realize chronic administration of ASO therapeutics to treat neurodegenerative diseases, new chemic...

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Main Authors: 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, Kristen Sanders, Jacob Stupalski, So Yasui, Diana Liu, Farid Benayoud, Hui Fang, Enxuan Jing, Makoto Ogo, Francis G. Fang, John Wang, Hyeong-wook Choi
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
tau
tauopathy
antisense oligonucleotides
PMO
gapmer
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124002919
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