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CRISPR-Cas systems for editing, regulating and targeting genomes
Open accessAbstract
Targeted genome editing using engineered nucleases has rapidly gone from being a niche technology to a mainstream method used by many biological researchers. This widespread adoption has been largely fueled by the emergence of the clustered, regularly interspaced, short palindromic repeat (CRISPR) technology, an important new approach for generating RNA-guided nucleases, such as Cas9, with customizable specificities. Genome editing mediated by these nucleases has been used to rapidly, easily and efficiently modify endogenous genes in a wide variety of biomedically important cell types and in organisms that have traditionally been challenging to manipulate genetically. Furthermore, a modified version of the CRISPR-Cas9 system has been developed to recruit heterologous domains that can regulate endogenous gene expression or label specific genomic loci in living cells. Although the genome-wide specificities of CRISPR-Cas9 systems remain to be fully defined, the power of these systems to perform targeted, highly efficient alterations of genome sequence and gene expression will undoubtedly transform biological research and spur the development of novel molecular therapeutics for human disease.
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“CRISPRs, a type of important repeat elements, are related to the adaptive immunity system that has been widely identified and characterized in many archaea and bacteria.63 , 64”
From Whole genomic DNA sequencing and comparative genomic analysis of Arthrospira platensis: high genome plasticity and genetic diversity (2016) · cites this paper as [64]
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“While complete gene knockout is often preferred, there are some drawbacks of CRISPR technology including Cas9 off-target activity [46], as well as the cost and labor-intensive process of guide RNA design, viral packaging and infection, isolation of single cells, expansion of clones, and verification of correct targeting [47].”
From Regulation of Enterocyte Brush Border Membrane Primary Na-Absorptive Transporters in Human Intestinal Organoid-Derived Monolayers (2024) · cites this paper as [46]
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“Additionally, targeted genome editing approaches such as CRISPR-Cas9 and related technologies may be used to actively introduce highly specific changes in the genome instead of screening for random changes (reviewed in Sander and Joung, 2014).”
From Shaping plant architecture (2015) · cites this paper as Sander and Joung, 2014
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Metadata
- Authors
- Sander, J. D., & Joung, J. K.
- Affiliations
- Harvard University (US)
- Corresponding author
- Joung, J Keith
- Venue
- Nature Biotechnology
- Published
- 2014-03-02
- Volume
- 32
- Issue
- 4
- Pages
- 347-355
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- PMC
- PMC4022601
- OpenAlex
- W2039705224
- Cited by
- 3,153
- References
- 102
- Citations by year
- 2026: 79 · 2025: 153 · 2024: 167 · 2023: 166 · 2022: 233 · 2021: 225 · 2020: 271 · 2019: 298 · 2018: 379 · 2017: 368 · 2016: 384 · 2015: 300 · 2014: 124
- FWCI
- 36.95
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institute of General Medical Sciences (DP1GM105378, R01GM088040)
- National Center for Chronic Disease Prevention and Health Promotion (DP1 GM105378)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- CRISPR/Cas9, genome editing, RNA-guided nucleases, engineered nucleases, gene expression regulation, targeted mutagenesis, molecular therapeutics
- MeSH terms
- Animals, Genetic Engineering, Humans, Rabbits, Genomics, Mice, Rats, Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR-Cas Systems
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