Brain-wide Genome Editing via STEP-RNPs for Treatment of Angelman Syndrome
Xiaona Lu, Ying Xie, Youmei Bao, Jiang Yu, Zefeng Wang, Jiali Fan, Wendy C. Sheu, Gretchen Long, Zewei Tu, Jie Tong, Garret Manquen, Yichao Li, Varun Katta, Ryan Dz-Wei Chow, Binfan Chen, , Jiangbing Zhou
This paper develops a non-viral genome-editing method called STEP-RNPs that effectively treats Angelman syndrome in mice and human neurons, showing functional genetic rescue without off-target effects.
bioRxiv · Nov 13, 2025 · PreprintGenetics