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Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing
Open accessAbstract
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.
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Metadata
- Authors
- 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.
- Venue
- Nature Communications
- Published
- 2018-06-14
- Volume
- 9
- Issue
- 1
- Pages
- 2338-2338
- ISSN
- 2041-1723 (ISSN-L)
- OpenAlex
- W2805837749
- Cited by
- 2,074
- References
- 5
- FWCI
- 158.36
- Type
- journal article
- Language
- en
- Open access
- gold
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