GFP-on mouse model for interrogation of in vivo gene editing.
| Authors | |
| Abstract | Gene editing technologies have revolutionized therapies for numerous genetic diseases. However, in vivo gene editing hinges on identifying efficient delivery vehicles for editing in targeted cell types, a significant hurdle in fully realizing its therapeutic potential. A model system to rapidly evaluate systemic gene editing would advance the field. Here, we develop the GFP-on reporter mouse, which harbors a nonsense mutation in a genomic EGFP sequence correctable by adenine base editor (ABE) among other genome editors. The GFP-on system was validated using single and dual adeno-associated virus (AAV9) encoding ABE8e and sgRNA. Intravenous administration of AAV9-ABE8e-sgRNA into adult GFP-on mice results in EGFP expression consistent with the tropism of AAV9. Intrahepatic delivery of AAV9-ABE8e-sgRNA into GFP-on fetal mice restores EGFP expression in AAV9-targeted organs lasting at least six months post-treatment. The GFP-on model provides an ideal platform for high-throughput evaluation of emerging gene editing tools and delivery modalities. |
| Year of Publication | 2025
|
| Journal | Nature communications
|
| Volume | 16
|
| Issue | 1
|
| Pages | 7017
|
| Date Published | 07/2025
|
| ISSN | 2041-1723
|
| DOI | 10.1038/s41467-025-61449-y
|
| PubMed ID | 40744920
|
| Links |




