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Fluorescent in vivo editing reporter (FIVER): A novel multispectral reporter of in vivo genome editing

By Peter A. Tennant, Robert G. Foster, Daniel O. Dodd, Ieng Fong Sou, Fraser McPhie, Nicholas Younger, Laura C Murphy, Matthew Pearson, Bertrand Vernay, Margaret A Keighren, Peter Budd, Stephen L. Hart, Roly Megaw, Luke Boulter, Pleasantine Mill

Posted 14 Jul 2020
bioRxiv DOI: 10.1101/2020.07.14.200170

Advances in genome editing technologies have created opportunities to treat rare genetic diseases, which are often overlooked in terms of therapeutic development. Nonetheless, substantial challenges remain: namely, achieving therapeutically beneficial levels and kinds of editing in the right cell type(s). Here we describe the development of FIVER (fluorescent in vivo editing reporter) - a modular toolkit for in vivo detection of genome editing with distinct fluorescent read-outs for non-homologous end-joining (NHEJ), homology-directed repair (HDR) and homology-independent targeted integration (HITI). We demonstrate that fluorescent outcomes reliably report genetic changes following editing with diverse genome editors in primary cells, organoids and in vivo . We show the potential of FIVER for high-throughput unbiased screens, from small molecule modulators of genome editing outcomes in primary cells through to genome-wide in vivo CRISPR cancer screens. Importantly, we demonstrate its in vivo application in postnatal organ systems of interest for genetic therapies - retina and liver. FIVER will broadly help expedite the development of therapeutic genome surgery for many genetic disorders. ### Competing Interest Statement The authors have declared no competing interest.

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