Article

Brain-wide Genome Editing via STEP-RNPs for Treatment of Angelman Syndrome

2025-11-14

Abstract excerpt

Brain-wide genome-editing remains a major hurdle for the treatment of neurogenetic disorders. Here, we report a non-viral, non-nanoparticle, chemical modification-based method, called Stimuli-Responsive Traceless Engineering Platform (STEP) that achieves highly efficient and brain-wide genome-editing of neurons in mice. Using cholesterol-based STEP as a lead, we show that a single administration of STEP-ribonucleo...

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Literature Corpus work: 0d2b29d6-1f2a-567a-8132-28a081dd31b2
DOI: 10.1101/2025.11.13.684643

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