| First Author | Song CQ | Year | 2020 |
| Journal | Nat Biomed Eng | Volume | 4 |
| Issue | 1 | Pages | 125-130 |
| PubMed ID | 31740768 | Mgi Jnum | J:285822 |
| Mgi Id | MGI:6400056 | Doi | 10.1038/s41551-019-0357-8 |
| Citation | Song CQ, et al. (2020) Adenine base editing in an adult mouse model of tyrosinaemia. Nat Biomed Eng 4(1):125-130 |
| abstractText | In contrast to traditional CRISPR-Cas9 homology-directed repair, base editing can correct point mutations without supplying a DNA-repair template. Here we show in a mouse model of tyrosinaemia that hydrodynamic tail-vein injection of plasmid DNA encoding the adenine base editor (ABE) and a single-guide RNA (sgRNA) can correct an A>G splice-site mutation. ABE treatment partially restored splicing, generated fumarylacetoacetate hydrolase (FAH)-positive hepatocytes in the liver, and rescued weight loss in mice. We also generated FAH(+) hepatocytes in the liver via lipid-nanoparticle-mediated delivery of a chemically modified sgRNA and an mRNA of a codon-optimized base editor that displayed higher base-editing efficiency than the standard ABEs. Our findings suggest that adenine base editing can be used for the correction of genetic diseases in adult animals. |
