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

ABSTRACT: 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 tdTomato in vivo genome-editing reporter (TIGER) 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 fluore

SUBMITTER: Du SW 

PROVIDER: S-EPMC12415246 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature