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In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

Cited 162 time in Web of Science Cited 177 time in Scopus
Authors
Kim, Eunji; Koo, Taeyoung; Park, Sung Wook; Kim, Daesik; Kim, Kyoungmi; Cho, Hee-Yeon; Song, Dong Woo; Lee, Kyu Jun; Jung, Min Hee; Kim, Seokjoong; Kim, Jin Hyoung; Kim, Jeong Hun; Kim, Jin-Soo
Issue Date
2017-02
Citation
Nature Communications, Vol.8, p. 14500
Abstract
Several CRISPR-Cas9 orthologues have been used for genome editing. Here, we present the smallest Cas9 orthologue characterized to date, derived from Campylobacter jejuni (CjCas9), for efficient genome editing in vivo. After determining protospacer-adjacent motif (PAM) sequences and optimizing single-guide RNA (sgRNA) length, we package the CjCas9 gene, its sgRNA sequence, and a marker gene in an all-in-one adeno-associated virus (AAV) vector and produce the resulting virus at a high titer. CjCas9 is highly specific, cleaving only a limited number of sites in the human or mouse genome. CjCas9, delivered via AAV, induces targeted mutations at high frequencies in mouse muscle cells or retinal pigment epithelium (RPE) cells. Furthermore, CjCas9 targeted to the Vegfa or Hif1a gene in RPE cells reduces the size of laser-induced choroidal neovascularization, suggesting that in vivo genome editing with CjCas9 is a new option for the treatment of age-related macular degeneration.
ISSN
2041-1723
URI
http://hdl.handle.net/10371/165685
DOI
https://doi.org/10.1038/ncomms14500
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