BROWSE

Related Scientist

GEL's photo.

GEL
유전체 교정 연구단
more info

ITEM VIEW & DOWNLOAD

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophyHighly Cited Paper

DC Field Value Language
dc.contributor.authorSeuk-Min Ryu-
dc.contributor.authorTaeyoung Koo-
dc.contributor.authorKyoungmi Kim-
dc.contributor.authorKayeong Lim-
dc.contributor.authorGayoung Baek-
dc.contributor.authorSang-Tae Kim-
dc.contributor.authorHeon Seok Kim-
dc.contributor.authorDa-eun Kim-
dc.contributor.authorHyunji Lee-
dc.contributor.authorEugene Chung-
dc.contributor.authorJin-Soo Kim-
dc.date.available2019-02-12T11:02:10Z-
dc.date.created2018-07-23-
dc.date.issued2018-06-
dc.identifier.issn1087-0156-
dc.identifier.urihttps://pr.ibs.re.kr/handle/8788114/5560-
dc.description.abstractAdenine 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-
dc.description.uri1-
dc.language영어-
dc.publisherNATURE PUBLISHING GROUP-
dc.titleAdenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy-
dc.typeArticle-
dc.type.rimsART-
dc.identifier.wosid000434689200022-
dc.identifier.scopusid2-s2.0-85048365258-
dc.identifier.rimsid64133-
dc.contributor.affiliatedAuthorSeuk-Min Ryu-
dc.contributor.affiliatedAuthorTaeyoung Koo-
dc.contributor.affiliatedAuthorKyoungmi Kim-
dc.contributor.affiliatedAuthorKayeong Lim-
dc.contributor.affiliatedAuthorGayoung Baek-
dc.contributor.affiliatedAuthorSang-Tae Kim-
dc.contributor.affiliatedAuthorHeon Seok Kim-
dc.contributor.affiliatedAuthorDa-eun Kim-
dc.contributor.affiliatedAuthorHyunji Lee-
dc.contributor.affiliatedAuthorEugene Chung-
dc.contributor.affiliatedAuthorJin-Soo Kim-
dc.identifier.doi10.1038/nbt.4148-
dc.identifier.bibliographicCitationNATURE BIOTECHNOLOGY, v.36, no.6, pp.536 - 539-
dc.citation.titleNATURE BIOTECHNOLOGY-
dc.citation.volume36-
dc.citation.number6-
dc.citation.startPage536-
dc.citation.endPage539-
dc.description.journalClass1-
dc.description.journalRegisteredClassscie-
dc.description.journalRegisteredClassscopus-
Appears in Collections:
Center for Genome Engineering(유전체 교정 연구단) > 1. Journal Papers (저널논문)
Files in This Item:
1806_류석민_NBT4148.pdfDownload

qrcode

  • facebook

    twitter

  • Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.
해당 아이템을 이메일로 공유하기 원하시면 인증을 거치시기 바랍니다.

Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.

Browse