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Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

Authors
Ryu, Seuk-MinKoo, TaeyoungKim, KyoungmiLim, KayeongBaek, GayoungKim, Sang-TaeKim, Heon SeokKim, Da-eunLee, HyunjiChung, EugeneKim, Jin-Soo
Issue Date
6월-2018
Publisher
NATURE PUBLISHING GROUP
Citation
NATURE BIOTECHNOLOGY, v.36, no.6, pp.536 - 539
Indexed
SCIE
SCOPUS
Journal Title
NATURE BIOTECHNOLOGY
Volume
36
Number
6
Start Page
536
End Page
539
URI
https://scholar.korea.ac.kr/handle/2021.sw.korea/74994
DOI
10.1038/nbt.4148
ISSN
1087-0156
Abstract
Adenine base editors (ABEs) composed of an engineered adenine deaminase and the Streptococcus pyogenes Cas9 nickase enable adenine-to-guanine (A-to-G) single-nucleotide substitutions in a guide RNA (gRNA)-dependent manner. Here we demonstrate application of this technology in mouse embryos and adult mice. We also show that long gRNAs enable adenine editing at positions one or two bases upstream of the window that is accessible with standard single guide RNAs (sgRNAs). We introduced the Himalayan point mutation in the Tyr gene by microinjecting ABE mRNA and an extended gRNA into mouse embryos, obtaining Tyr mutant mice with an albino phenotype. Furthermore, we delivered the split ABE gene, using trans-splicing adenoassociated viral vectors, to muscle cells in a mouse model of Duchenne muscular dystrophy to correct a nonsense mutation in the Dmd gene, demonstrating the therapeutic potential of base editing in adult animals.
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