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.
journal_name
Nat Communjournal_title
Nature communicationsauthors
Kim E,Koo T,Park SW,Kim D,Kim K,Cho HY,Song DW,Lee KJ,Jung MH,Kim S,Kim JH,Kim JH,Kim JSdoi
10.1038/ncomms14500subject
Has Abstractpub_date
2017-02-21 00:00:00pages
14500issn
2041-1723pii
ncomms14500journal_volume
8pub_type
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