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유전체교정 연구단
유전체 교정 연구단
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Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

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Title
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
Author(s)
Seuk-Min Ryu; Taeyoung Koo; Kyoungmi Kim; Kayeong Lim; Gayoung Baek; Sang-Tae Kim; Heon Seok Kim; Da-eun Kim; Hyunji Lee; Eugene Chung; Jin-Soo Kim
Publication Date
2018-06
Journal
NATURE BIOTECHNOLOGY, v.36, no.6, pp.536 - 539
Publisher
NATURE PUBLISHING GROUP
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 adeno-associated 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. © 2018 Nature America, Inc., part of Springer Nature. All rights reserved
URI
https://pr.ibs.re.kr/handle/8788114/5560
ISSN
1087-0156
Appears in Collections:
Center for Genome Engineering(유전체 교정 연구단) > Journal Papers (저널논문)
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