TY - JOUR T1 - Single AAV-mediated scarless genome editing in dysfunctional retinal neurons mediates robust visual restoration in mice JF - bioRxiv DO - 10.1101/552653 SP - 552653 AU - Koji M Nishiguchi AU - Kosuke Fujita AU - Shota Katayama AU - Toru Nakazawa Y1 - 2019/01/01 UR - http://biorxiv.org/content/early/2019/02/25/552653.abstract N2 - The most versatile treatment for inherited disorders is to precisely replace a mutated sequence with its wildtype counterpart, thereby “normalizing” the genome. We developed a single AAV platform that allows this in retinal neurons with combined CRISPR-Cas9 and micro-homology-mediated end-joining. In blind mice, the platform rescued ~10% of the retinal neurons, resulting in an incredible ~10,000-fold improvement in light sensitivity, equivalent to the restoration mediated by conventional gene augmentation therapy. ER -