{"id":"doi:10.1038/s41467-018-04768-7","kind":"article","title":"Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing","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"],"author_count":13,"year":2018,"published":"2018-06-14","venue":"Nature communications","cited_by":120,"doi":"10.1038/s41467-018-04768-7","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41467-018-04768-7","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41467-018-04768-7","updated":null,"pmid":"29904106","pmcid":"PMC6002399","openalex_id":null,"pdf_url":"https://europepmc.org/articles/PMC6002399/pdf","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_license":"CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)","license":"CC-BY","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":[],"details":null,"provenance":"europepmc","attribution":"Abstract of \"Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing\" by Liu Z, Lu Z, Yang G et al., https://doi.org/10.1038/s41467-018-04768-7, used under its Creative Commons Attribution license (CC-BY); markup removed, otherwise unmodified.","topics":[{"slug":"gene-editing-therapies","name":"CRISPR & Gene-Editing Therapies","domain_slug":"medicine"}],"cited_in_dossiers":[]}