The DOI matches its Crossref record; 34 of the 34 references it cites were found in a registry or an index.
Genome Engineering with Targetable Nucleases
Abstract
Current technology enables the production of highly specific genome modifications with excellent efficiency and specificity. Key to this capability are targetable DNA cleavage reagents and cellular DNA repair pathways. The break made by these reagents can produce localized sequence changes through inaccurate nonhomologous end joining (NHEJ), often leading to gene inactivation. Alternatively, user-provided DNA can be used as a template for repair by homologous recombination (HR), leading to the introduction of desired sequence changes. This review describes three classes of targetable cleavage reagents: zinc-finger nucleases (ZFNs), transcription activator–like effector nucleases (TALENs), and CRISPR/Cas RNA-guided nucleases (RGNs). As a group, these reagents have been successfully used to modify genomic sequences in a wide variety of cells and organisms, including humans. This review discusses the properties, advantages, and limitations of each system, as well as the specific considerations required for their use in different biological systems.
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“Genome-editing technologies may offer a powerful approach to treat many human diseases, including HIV/AIDS, haemophilia, sickle-cell anaemia and several forms of cancer 3 .”
From Don’t edit the human germ line (2015) · cites this paper as [3]
Citations
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Induced Pluripotent Stem Cells Meet Genome Editing
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“The low frequency of spontaneous homology mediated gene targeting in hPSCs was dramatically increased through the development of site-specific nucleases (SSN) as a tool for their genetic engineering (reviewed in (Carroll, 2014; Hsu et al., 2014; Urnov et al., 2010)).”
MethodDon’t edit the human germ line
Lanphier, E., Urnov, F., Haecker, S. E., Werner, M., & Smolenski, J. (2015) · Nature · doi:10.1038/519410a · Cited by 530
Metadata
- Authors
- Carroll, D.
- Affiliations
- University of Utah (US)
- Corresponding author
- Carroll, Dana
- ORCID
- Carroll, Dana
- Venue
- Annual Review of Biochemistry
- Publisher
- Annual Reviews
- Published
- 2014-06-02
- Volume
- 83
- Issue
- 1
- Pages
- 409-439
- ISSN
- 0066-4154 (ISSN-L)
- 1545-4509
- OpenAlex
- W2123366092
- Cited by
- 568
- References
- 34
- Citations by year
- 2026: 16 · 2025: 29 · 2024: 46 · 2023: 33 · 2022: 35 · 2021: 45 · 2020: 40 · 2019: 43 · 2018: 52 · 2017: 60 · 2016: 64 · 2015: 77 · 2014: 27
- FWCI
- 6.84
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Annual Review of Biochemistry (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institute of General Medical Sciences (R01GM078571)
- 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 › DNA Repair Mechanisms
- Keywords
- genome editing, zinc finger nucleases, TALEN, CRISPR-Cas, RNA-guided nucleases, DNA repair, non-homologous end joining, homologous recombination, gene inactivation
- MeSH terms
- Animals, DNA, DNA Damage, DNA Repair, Drosophila, Drosophila melanogaster, Endonucleases, Genetic Engineering, Humans, Recombination, Genetic, Zebrafish, Protein Engineering, Zinc Fingers, Genome, Gene Deletion, Arabidopsis, Protein Structure, Tertiary, Genomics, Mice, Rats, DNA End-Joining Repair
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