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

Eunji Kim, Taeyoung Koo, Sung Wook Park, Daesik Kim, Kyoungmi Kim, Hee-Yeon Cho, Dong Woo Song, Kyu Jun Lee, Min Hee Jung, Seokjoong Kim, Jin Hyoung Kim, Jeong Hun Kim () and Jin-Soo Kim ()
Additional contact information
Eunji Kim: Center for Genome Engineering, Institute for Basic Science (IBS)
Taeyoung Koo: Center for Genome Engineering, Institute for Basic Science (IBS)
Sung Wook Park: Seoul National University College of Medicine
Daesik Kim: Center for Genome Engineering, Institute for Basic Science (IBS)
Kyoungmi Kim: Center for Genome Engineering, Institute for Basic Science (IBS)
Hee-Yeon Cho: Center for Genome Engineering, Institute for Basic Science (IBS)
Dong Woo Song: ToolGen, Byucksan Digital Valley 6-cha
Kyu Jun Lee: ToolGen, Byucksan Digital Valley 6-cha
Min Hee Jung: ToolGen, Byucksan Digital Valley 6-cha
Seokjoong Kim: ToolGen, Byucksan Digital Valley 6-cha
Jin Hyoung Kim: Seoul National University College of Medicine
Jeong Hun Kim: Seoul National University College of Medicine
Jin-Soo Kim: Center for Genome Engineering, Institute for Basic Science (IBS)

Nature Communications, 2017, vol. 8, issue 1, 1-12

Abstract: 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.

Date: 2017
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DOI: 10.1038/ncomms14500

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