• Publications
  • Influence
Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins.
TLDR
Delivery of purified recombinant Cas9 protein and guide RNA into cultured human cells including hard-to-transfect fibroblasts and pluripotent stem cells is delivered and RGEN ribonucleoproteins (RNPs) induce site-specific mutations at frequencies of up to 79%, while reducing off- target mutations associated with plasmid transfection at off-target sites. Expand
Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells
TLDR
It is found that Cpf1 could tolerate single or double mismatches in the 3′ Pam-distal region, but not in the 5′ PAM-proximal region, and off-target effects were completely abrogated by using preassembled, recombinant CpF1 ribonucleoproteins. Expand
In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni
TLDR
CjCas9, delivered via AAV, induces targeted mutations at high frequencies in mouse muscle cells or retinal pigment epithelium (RPE) cells, and reduces the size of laser-induced choroidal neovascularization, suggesting that in vivo genome editing with Cj Cas9 is a new option for the treatment of age-related macular degeneration. Expand
Correction of a pathogenic gene mutation in human embryos
Genome-wide target specificities of CRISPR RNA-guided programmable deaminases
TLDR
It is found that the rAPOBEC1–nCas9 base editor is highly specific, inducing cytosine-to-uracil conversions at only 18 ± 9 sites in the human genome for each sgRNA. Expand
Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells
TLDR
Digenome-seq is a robust, sensitive, unbiased and cost-effective method for profiling genome-wide off-target effects of programmable nucleases including Cas9, and shows that Cas9 off- target effects can be avoided by replacing 'promiscuous' single guide RNAs (sgRNAs) with modified sgRNAs. Expand
Highly efficient RNA-guided base editing in mouse embryos
TLDR
Base editors composed of a cytidine deaminase fused to CRISPR–Cas9 convert cytidine to uridine, leading to single-base-pair substitutions in eukaryotic cells, demonstrating an efficient method to generate mice with targeted point mutations. Expand
Genotyping with CRISPR-Cas-derived RNA-guided endonucleases.
TLDR
A novel method of employing CRISPR/Cas-derived RNA-guided engineered nucleases (RGENs) in RFLP analysis that can detect homozygous mutant clones that contain identical biallelic indel sequences and is not limited by sequence polymorphisms near the nuclease target sites is presented. Expand
Genome-wide target specificities of CRISPR-Cas9 nucleases revealed by multiplex Digenome-seq.
TLDR
It is found that many false-positive, bulge-type off-target sites were cleaved by sgRNAs transcribed from an oligonucleotide duplex but not by those transcribes from a plasmid template. Expand
Correction of a pathogenic gene mutation in human embryos
TLDR
The efficiency, accuracy and safety of the approach presented suggest that it has potential to be used for the correction of heritable mutations in human embryos by complementing preimplantation genetic diagnosis. Expand
...
1
2
3
4
5
...