GeneticsbioRxivPreprint
Brain-wide Genome Editing via STEP-RNPs for Treatment of Angelman Syndrome
Xiaona Lu, Ying Xie, Youmei Bao…·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.