Treating Myotonic Dystrophy with artificial RNA endonucleases to specifically degrade toxic RNA expansions
Tong Wei, Hanyang Hu, Xin Jiang, Yucheng Li, Wenjian Han, Yi Yang, Xiao Xiao, Miaowei Mao, Zefeng Wang
This paper develops artificial RNA endonucleases (ARCs) that specifically degrade toxic RNA expansions in DM1, showing efficacy in cells and mouse models with low immunogenicity and off-target effects.
bioRxiv · Jun 10, 2025 · PreprintSynthetic Biology