# Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing

Article · Liu Z, Lu Z, Yang G, Huang S, Li G, Feng S, Liu Y, Li J, Yu W, Zhang Y, Chen J, Sun Q, Huang X

- Archive id: doi:10.1038/s41467-018-04768-7
- Published: 2018-06-14
- Venue: Nature communications
- DOI: https://doi.org/10.1038/s41467-018-04768-7
- PMID: 29904106
- PMCID: PMC6002399
- Citations: 120
- Source URL: https://doi.org/10.1038/s41467-018-04768-7
- PDF: https://europepmc.org/articles/PMC6002399/pdf
- Provenance: europepmc

## Abstract

A recently developed adenine base editor (ABE) efficiently converts A to G and is potentially useful for clinical applications. However, its precision and efficiency in vivo remains to be addressed. Here we achieve A-to-G conversion in vivo at frequencies up to 100% by microinjection of ABE mRNA together with sgRNAs. We then generate mouse models harboring clinically relevant mutations at Ar and Hoxd13, which recapitulates respective clinical defects. Furthermore, we achieve both C-to-T and A-to-G base editing by using a combination of ABE and SaBE3, thus creating mouse model harboring multiple mutations. We also demonstrate the specificity of ABE by deep sequencing and whole-genome sequencing (WGS). Taken together, ABE is highly efficient and precise in vivo, making it feasible to model and potentially cure relevant genetic diseases.

_Abstract reproduced under CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)._

Topics: [CRISPR & Gene-Editing Therapies](https://space-kitty.com/topics/gene-editing-therapies)

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