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タイトル
  • ja Efficient viral delivery of Cas9 into human safe harbor
作成者
    • Hayashi, Hideki
    • Kubo, Yoshinao
    • Izumida, Mai
    • Matsuyama, Toshifumi
権利情報
  • c 2020, The Author(s). Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
内容注記
  • Other Gene editing using CRISPR/Cas9 is a promising method to cure many human genetic diseases. We have developed an efficient system to deliver Cas9 into the adeno-associated virus integration site 1 (AAVS1) locus, known as a safe harbor, using lentivirus and AAV viral vectors, as a step toward future in vivo transduction. First, we introduced Cas9v1 (derived from Streptococcus pyogenes) at random into the genome using a lentiviral vector. Cas9v1 activity was used when the N-terminal 1.9 kb, and C-terminal 2.3 kb fragments of another Cas9v2 (human codon-optimized) were employed sequentially with specific single-guide RNAs (sgRNAs) and homology donors carried by AAV vectors into the AAVS1 locus. Then, Cas9v1 was removed from the genome by another AAV vector containing sgRNA targeting the long terminal repeat of the lentivirus vector. The reconstituted Cas9v2 in the AAVS1 locus was functional and gene editing was efficient.
  • Other identifier:Scientific Reports, 10(1), art.no.21474; 2020
出版者 Springer Nature
日付
    Created2020-12-24 , Issued2020-12-08
言語
  • eng
資源識別のタイプ journal article
出版タイプ VoR
資源識別子 URI http://hdl.handle.net/10069/40451
関連
  • isIdenticalTo DOI https://doi.org/10.1038/s41598-020-78450-8
収録誌情報
    • ISSN 2045-2322
      • Scientific Reports 10(1), 21474
ファイル
コンテンツ更新日時 2021-02-18