Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope

Tingting Jiang, Jordana M. Henderson, Kevin Coote, Yi Cheng, Hillary C. Valley, Xiao-Ou Zhang, Qin Wang, Luke H. Rhym, Yueying Cao, Gregory A. Newby, Hermann Bihler, Martin Mense, Zhiping Weng, Daniel G. Anderson, Anton P. McCaffrey, David R. Liu and Wen Xue ()
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Tingting Jiang: University of Massachusetts Medical School
Jordana M. Henderson: TriLink BioTechnologies
Kevin Coote: Cystic Fibrosis Foundation, CFFT Lab
Yi Cheng: Cystic Fibrosis Foundation, CFFT Lab
Hillary C. Valley: Cystic Fibrosis Foundation, CFFT Lab
Xiao-Ou Zhang: University of Massachusetts Medical School
Qin Wang: Tongji University
Luke H. Rhym: Massachusetts Institute of Technology
Yueying Cao: University of Massachusetts Medical School
Gregory A. Newby: Broad Institute of Harvard and MIT
Hermann Bihler: Cystic Fibrosis Foundation, CFFT Lab
Martin Mense: Cystic Fibrosis Foundation, CFFT Lab
Zhiping Weng: University of Massachusetts Medical School
Daniel G. Anderson: Massachusetts Institute of Technology
Anton P. McCaffrey: TriLink BioTechnologies
David R. Liu: Broad Institute of Harvard and MIT
Wen Xue: University of Massachusetts Medical School

Nature Communications, 2020, vol. 11, issue 1, 1-9

Abstract: Abstract CRISPR-Cas9-associated base editing is a promising tool to correct pathogenic single nucleotide mutations in research or therapeutic settings. Efficient base editing requires cellular exposure to levels of base editors that can be difficult to attain in hard-to-transfect cells or in vivo. Here we engineer a chemically modified mRNA-encoded adenine base editor that mediates robust editing at various cellular genomic sites together with moderately modified guide RNA, and show its therapeutic potential in correcting pathogenic single nucleotide mutations in cell and animal models of diseases. The optimized chemical modifications of adenine base editor mRNA and guide RNA expand the applicability of CRISPR-associated gene editing tools in vitro and in vivo.

Date: 2020
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DOI: 10.1038/s41467-020-15892-8

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