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Genome editing using RNA-guided nucleases in their ribonucleoprotein (RNP) form represents a promising strategy for gene modification and therapy because they are free of exogenous DNA integration and have reduced toxicity in vivo and ex vivo. However, genome editing by Cas9 nuclease from Staphylococcus aureus (SaCas9), has not been reported in its RNP form, which recognizes a longer protospacer adjacent motif (PAM), 5'-NNGRRT-3', compared with SpCas9(Streptococcus pyogenes Cas9) of 5'-NGG-3' PAM. Here, we report SaCas9-RNP mediated genome editing in human cells. The SaCas9-RNP displayed efficient genome editing activities of enhanced green fluorescent protein (EGFP) coding gene as well as three endogenous genes (OPA1, RS1 and VEGFA). We further successfully implemented SaCas9-RNP to correct a pathogenic RS1 mutation for X-linked Juvenile Retinoschisis. We also showed that off-target effects triggered by SaCas9-RNP were undetectable by targeted deep sequencing. Collectively, this study demonstrates the potential of SaCas9-RNP mediated genome editing in human cells, which could facilitate genome editing based therapy. This article is protected by copyright. All rights reserved.
This article was published in the following journal.
Name: Biotechnology journal
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system discovered using bacteria has been repurposed for genome editing in human cells. Transient express...
The CRISPR- Streptococcus pyogenes Cas9 (SpCas9) system offers a rapid, simple and flexible genome editing approach. However, the targeting scope of the SpCas9 system is limited by the canonical NGG P...
The great advances in exploiting the CRISPR/Cas9 system are paving the way for targeted genome engineering in plants. Genome editing by direct delivery of CRISPR/Cas9 ribonucleoprotein complexes (RNPs...
Marchantia polymorpha is one of the model species of basal land plants. Although CRISPR/Cas9-based genome editing has already been demonstrated for this plant, the efficiency was too low to apply to f...
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Component of the NATIONAL INSTITUTES OF HEALTH. It conducts and supports research into the mapping of the human genome and other organism genomes. The National Center for Human Genome Research was established in 1989 and re-named the National Human Genome Research Institute in 1997.
The complete genetic complement contained in the DNA of a set of CHROMOSOMES in a HUMAN. The length of the human genome is about 3 billion base pairs.
A coordinated effort of researchers to map (CHROMOSOME MAPPING) and sequence (SEQUENCE ANALYSIS, DNA) the human GENOME.
Genetic engineering techniques that involve DNA REPAIR mechanisms for incorporating site-specific modifications into a cell's genome.
The type species of BETACORONAVIRUS genus causing gastroenteritis respiratory diseases in mammals. Previously separate species HUMAN CORONAVIRUS OC43; BOVINE CORONAVIRUS; Human enteric coronavirus; Equine coronavirus; and Porcine hemagglutinating encephalomyelitis virus merged into this species on the basis of similar genome nucleotide sequence and genome organization.
Bioinformatics is the application of computer software and hardware to the management of biological data to create useful information. Computers are used to gather, store, analyze and integrate biological and genetic information which can then be applied...
Within medicine, nutrition (the study of food and the effect of its components on the body) has many different roles. Appropriate nutrition can help prevent certain diseases, or treat others. In critically ill patients, artificial feeding by tubes need t...
DNA sequencing is the process of determining the precise order of nucleotides within a DNA molecule. During DNA sequencing, the bases of a small fragment of DNA are sequentially identified from signals emitted as each fragment is re-synthesized from a ...