Single AAV-mediated scarless genome editing in dysfunctional retinal neurons mediates robust visual restoration in mice
DOI
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説明
<jats:p>The most versatile treatment for inherited disorders is to precisely replace a mutated sequence with its wildtype counterpart, thereby “<jats:italic>normalizing</jats:italic>” 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.</jats:p>