In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni
Institute for Basic Science · ToolGen (South Korea) · +3 more institutions
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…
Citation impact
- FWCI
- 31.06
- Percentile
- 100%
- References
- 39
Authors
13- EKEunji KimCorresponding
Institute for Basic Science, ToolGen (South Korea)
- TKTaeyoung Koo
Institute for Basic Science, Korea University of Science and Technology
- SWSung Wook Park
Seoul National University, Seoul National University Hospital
- DKDaesik Kim
Seoul National University, Institute for Basic Science
- KKKyoungmi Kim
Institute for Basic Science
Topics & keywords
- Biology
- Genome editing
- Cas9
- Campylobacter jejuni
- Genome
- CRISPR
- Subgenomic mRNA
- Gene