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タイトル: | Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR-Cas platform. |
著者: | Yoshimi, K Kaneko, T Voigt, B Mashimo, T |
著者名の別形: | 吉見, 一人 金子, 武人 真下, 知士 |
キーワード: | Biological sciences Biotechnology Genetics |
発行日: | 26-Jun-2014 |
出版者: | Nature Publishing Group |
誌名: | Nature communications |
巻: | 5 |
論文番号: | 4240 |
抄録: | The bacterial CRISPR/Cas system has proven to be an efficient gene-targeting tool in various organisms. Here we employ CRISPR/Cas for accurate and efficient genome editing in rats. The synthetic chimeric guide RNAs (gRNAs) discriminate a single-nucleotide polymorphism (SNP) difference in rat embryonic fibroblasts, allowing allele-specific genome editing of the dominant phenotype in (F344 × DA)F1 hybrid embryos. Interestingly, the targeted allele, initially assessed by the allele-specific gRNA, is repaired by an interallelic gene conversion between homologous chromosomes. Using single-stranded oligodeoxynucleotides, we recover three recessive phenotypes: the albino phenotype by SNP exchange; the non-agouti phenotype by integration of a 19-bp DNA fragment; and the hooded phenotype by eliminating a 7, 098-bp insertional DNA fragment, evolutionary-derived from an endogenous retrovirus. Successful in vivo application of the CRISPR/Cas system confirms its importance as a genetic engineering tool for creating animal models of human diseases and its potential use in gene therapy. |
記述: | CRISPR/Casシステムを用いたアレル特異的ゲノム編集とラット毛色突然変異の修復!.京都大学プレスリリース. 2014-06-27. |
著作権等: | This work is licensed under a Creative Commons Attribution 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/ |
URI: | http://hdl.handle.net/2433/188742 |
DOI(出版社版): | 10.1038/ncomms5240 |
PubMed ID: | 24967838 |
関連リンク: | https://www.kyoto-u.ac.jp/ja/research-news/2014-06-26 |
出現コレクション: | 学術雑誌掲載論文等 |
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