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Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors mediate marked visual restoration

NISHIGUCHI, K. M.; Fujita, K.; Miya, F.; Katayama, S.; Nakazawa, T.

2019-12-01 bioengineering
10.1101/552653 bioRxiv
Show abstract

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.

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"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.