TIGER: A tdTomato in-vivo genome-editing reporter mouse for investigating precision-editor delivery approaches

In vivo genome editing has the potential to address many inherited and environmental disorders. However, a major hurdle for the clinical translation of genome editing is safe, efficient delivery to disease-relevant tissues. A modality-agnostic reporter animal model that facilitates rapid, precise, and quantifiable assessment of functional delivery and editing could greatly enhance the evaluation and translation of delivery technologies. Here, we present the development of the TIGER (tdTomato in vivo genome-editing reporter) mouse, a reporter strain that harbors an integrated and constitutively expressed mutated tdTomato gene in the Polr2a locus. The mutations (Q115X, Q357X) abolish fluorescence, but successful adenine base editing (ABE) or prime editing (PE) restores tdTomato fluorescence. This mouse model facilitates the tissue- and cell-type-specific assessment of genome editing agent delivery. We describe several editing strategies validated in vitro and demonstrate efficient ABE and..., , # TIGER: A tdTomato in-vivo genome-editing reporter mouse for investigating precision-editor delivery approaches

Dataset DOI: [10.5061/dryad.866t1g23p](10.5061/dryad.866t1g23p)

## Description of the data and file structure

In vitro and in vivo evaluation of base and prime editing approaches for the characterization of a new tdTomato gene editing reporter mouse.

### Files and variables

* Figure_1.xlsx
* 1E: treatment vs. NGS reads with indicated edit (%)
* A7: only target adenine (position 7 in protospacer) edited
* A7+ A9: target adenine and bystander adenine (positions 7 and 9 in protospacer) edited
* Indel: insertions/deletions (unintended edits)
* Figure_2.xlsx
* 2E: tissue vs. ln(CPM) from scRNAseq (AU)
* Normalized count per million bp (CPM) from single cell RNA sequencing
* Figure_3.xlsx
* 3B: treatment vs. wavelength (nm) vs. fluorescence (AU)
* eVLP: engineered virus-like particle delivering base editor protein
* 3E: tissue location vs. wavelengt...,