Cited by 3,153CRISPR and Genetic EngineeringRNA regulation and diseaseRNA Interference and Gene Delivery

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CRISPR-Cas systems for editing, regulating and targeting genomes

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Sander, Jeffry D, Joung, J Keith·2014·Nature Biotechnology

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Abstract

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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Authors: Sander, J. D., & Joung, J. K.
Affiliations: Harvard University (US)
Corresponding author: Joung, J Keith
ORCID: Sander, Jeffry D
Joung, J Keith
Venue: Nature Biotechnology
Publisher: Springer Science and Business Media LLC
Published: 2014-03-02
Volume: 32
Issue: 4
Pages: 347-355
ISSN: 0733-222X (ISSN-L)
1087-0156
1546-1696
DOI: 10.1038/nbt.2842
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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