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Genome-Editing Technologies: Principles and Applications
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TALENs are composed of DNA binding domain and restriction domain like ZFN but their DNA binding domain has more potential target sequence than the ZFN gene-editing tool.
“TALENs are also composed of DNA binding domain and restriction domain like ZFN but their DNA binding domain has more potential target sequence than the ZFN gene-editing tool.”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [6]
In both ZFN and TALENs, the difficulty of protein engineering, being expensive, and time-consuming were major challenges for researchers and manufacturers.
“In both cases, the difficulty of protein engineering, being expensive, and time-consuming were the major challenges for researchers and manufacturers.6,7”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [6]
ZFN has a zinc finger DNA binding domain used to bind a specific target DNA sequence and a restriction endonuclease domain used to cleave the DNA at the target site.
“ZFN has a zinc finger DNA binding domain used to bind a specific target DNA sequence and a restriction endonuclease domain used to cleave the DNA at the target site.”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [6]
Before the discovery of CRISPR/Cas-9, scientists relied on two gene-editing techniques: zinc finger nucleases (ZFN) and Transcription activator-like effector nucleases (TALENs).
“Before the discovery of CRISPR/Cas-9, scientists were relied on two gene-editing techniques using restriction enzymes, zinc finger nucleases (ZFN) and Transcription activator-like effector nucleases (TALENs).6”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [6]
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“Because of the exquisite ease with which it can be programmed, the CRISPR-Cas9 system has also been adapted for transcriptional modulation through fusion of specific effector domains to a catalytically inactivated variant of the Cas9 protein (Qi et al. 2013).”
BackgroundDNA targeting specificity of RNA-guided Cas9 nucleases
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“…gene expression by simply blocking transcriptional initiation or elongation through a process known as CRISPR interference (Qi et al. 2013), although fusing dCas9 to transcriptional repressor domains can also lead to efficient silencing from the promoter (Gilbert et al. 2013; Zalatan et al. 2015).”
BackgroundHigh-Frequency off-Target Mutagenesis Induced by CRISPR-Cas Nucleases in Human Cells
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Efficient genome editing in zebrafish using a CRISPR-Cas system
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“Importantly, because of the ease with which the CRISPRCas9 system can be used, genome-wide screens using Cas9 transcriptional activators (Gilbert et al. 2014; Konermann et al. 2015) and repressors (Gilbert et al.”
BackgroundGenome-Scale CRISPR-Mediated Control of Gene Repression and Activation
Gilbert, L. A., Horlbeck, M. A., Adamson, B., Villalta, J. E., Chen, Y., Whitehead, E. H., Guimaraes, C., Panning, B., Ploegh, H. L., Bassik, M. C., et al. (2014) · Cell · doi:10.1016/j.cell.2014.09.029 · Cited by 2,728
In vivo genome editing using Staphylococcus aureus Cas9
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RNA-guided editing of bacterial genomes using CRISPR-Cas systems
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Crystal structure of Cas9 in complex with guide RNA and target DNA
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Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
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“2013), which increase gene-editing specificity by requiring the induction of two sequential and adjacent nicking events for DSB formation, or truncated gRNA that are more sensitive to mismatches at the genomic target site than a full-length gRNA (Fu et al. 2014).”
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RNA-guided genetic silencing systems in bacteria and archaea
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“To this end, considerable effort has been devoted to improving the specificity of this system, including using paired Cas9 nickases (Mali et al. 2013a; Ran et al. 2013), which increase gene-editing specificity by requiring the induction of two sequential and adjacent nicking events for DSB formation, or truncated gRNA…”
BackgroundMethodInfluentialEngineered CRISPR-Cas9 nucleases with altered PAM specificities
Kleinstiver, B. P., Prew, M. S., Tsai, S. Q., Topkar, V. V., Nguyen, N. T., Zheng, Z., Gonzales, A. P. W., Li, Z., Peterson, R. T., Yeh, J.-R. J., et al. (2015) · Nature · doi:10.1038/nature14592 · Cited by 1,624
Identification and characterization of essential genes in the human genome
Wang, T., Birsoy, K., Hughes, N. W., Krupczak, K. M., Post, Y., Wei, J. J., Lander, E. S., & Sabatini, D. M. (2015) · Science · doi:10.1126/science.aac7041 · Cited by 1,617
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Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
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Bibikova, M., Golic, M., Golic, K. G., & Carroll, D. (2002) · Genetics · doi:10.1093/genetics/161.3.1169 · Cited by 860
Chimeric nucleases stimulate gene targeting in human cells
Porteus, M. H., & Baltimore, D. (2003) · Science · doi:10.1126/science.1078395 · Cited by 741
CRISPR-mediated adaptive immune systems in bacteria and archaea
Sorek, R., Lawrence, C. M., & Wiedenheft, B. (2013) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-072911-172315 · Cited by 668
Correction of a genetic disease in mouse via use of CRISPR-Cas9
Wu, Y., Liang, D., Wang, Y., Bai, M., Tang, W., Bao, S., Yan, Z., Li, D., & Li, J. (2013) · Cell stem cell · doi:10.1016/j.stem.2013.10.016 · Cited by 614 · cited 1×
“…the rapid creation of transgenic zebrafish (Doyon et al. 2008; Sander et al. 2011a; Hwang et al. 2013), mice (Cui et al. 2011; Wang et al. 2013; Wu et al. 2013), rats (Geurts et al. 2009; Tesson et al. 2011; Li et al. 2013), monkeys (Liu et al. 2014c), and livestock (Hauschild et al. 2011;…”
BackgroundA dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Yang, Y., Wang, L., Bell, P., McMenamin, D., He, Z., White, J., Yu, H., Xu, C., Morizono, H., Musunuru, K., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3469 · Cited by 556 · cited 2×
“…particle AAV system, wherein one AAV vector carried the Cas9 nuclease and a second harbored the gRNA and donor repair template, was able to mediate correction of a disease-causing mutation in the ornithine transcarbamylase gene in the liver of a neonatal model of the disease (Yang et al. 2016).”
BackgroundIn vivo genome editing restores haemostasis in a mouse model of haemophilia
Li, H., Haurigot, V., Doyon, Y., Li, T., Wong, S. Y., Bhagwat, A. S., Malani, N., Anguela, X. M., Sharma, R., Ivanciu, L., et al. (2011) · Nature · doi:10.1038/nature10177 · Cited by 548
Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures
Doyon, Y., Vo, T. D., Mendel, M. C., Greenberg, S. G., Wang, J., Xia, D. F., Miller, J. C., Urnov, F. D., Gregory, P. D., & Holmes, M. C. (2011) · Nature Methods · doi:10.1038/nmeth.1539 · Cited by 402
Holkers, M., Maggio, I., Liu, J., Janssen, J. M., Miselli, F., Mussolino, C., Recchia, A., Cathomen, T., & Gonçalves, M. A. F. V. (2013) · Nucleic Acids Research · doi:10.1093/nar/gks1446 · Cited by 312
Self-citationTargeted gene knockout by direct delivery of zinc-finger nuclease proteins
Gaj, T., Guo, J., Kato, Y., Sirk, S. J., & Barbas, C. F. (2012) · Nature Methods · doi:10.1038/nmeth.2030 · Cited by 306
Self-citationLiu, Y., Zeng, Y., Liu, L., Zhuang, C., Fu, X., Huang, W., & Cai, Z. (2014) · Nature Communications · doi:10.1038/ncomms6393 · Cited by 223 · cited 4×
“…et al. 2013), mice (Cui et al. 2011; Wang et al. 2013; Wu et al. 2013), rats (Geurts et al. 2009; Tesson et al. 2011; Li et al. 2013), monkeys (Liu et al. 2014c), and livestock (Hauschild et al. 2011; Carlson et al. 2012), which together have the capacity to accelerate human disease modeling…”
BackgroundInfluentialSather, B. D., Romano Ibarra, G. S., Sommer, K., Curinga, G., Hale, M., Khan, I. F., Singh, S., Song, Y., Gwiazda, K., Sahni, J., et al. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aac5530 · Cited by 223
In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency
Mahiny, A. J., Dewerth, A., Mays, L. E., Alkhaled, M., Mothes, B., Malaeksefat, E., Loretz, B., Rottenberger, J., Brosch, D. M., Reautschnig, P., et al. (2015) · Nature Biotechnology · doi:10.1038/nbt.3241 · Cited by 139 · cited 2×
“Methods for overcoming these limitations have emerged as TALENs can be readily delivered into cells as mRNA (Mahiny et al. 2015; Mock et al. 2015) and even protein (Cai et al. 2014; Liu et al. 2014a), although alternative codon usage and amino acid degeneracy can also be leveraged to express RVD…”
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200 of 219 shown
Cited by
Sorted by citations
The Post-GWAS Era: From Association to Function
Gallagher, M. D., & Chen-Plotkin, A. S. (2018) · The American Journal of Human Genetics · doi:10.1016/j.ajhg.2018.04.002 · Cited by 822
Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing
Mengstie, M. A., & Wondimu, B. Z. (2021) · Biologics: Targets and Therapy · doi:10.2147/btt.s326422 · Cited by 478
In vivo genome editing improves motor function and extends survival in a mouse model of ALS
Gaj, T., Ojala, D. S., Ekman, F. K., Byrne, L. C., Limsirichai, P., & Schaffer, D. V. (2017) · Science Advances · doi:10.1126/sciadv.aar3952 · Cited by 185
Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions
Drag, S., Dotiwala, F., & Upadhyay, A. K. (2023) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.64.7.39 · Cited by 145
Gaj, T., Staahl, B. T., Rodrigues, G. M. C., Limsirichai, P., Ekman, F. K., Doudna, J. A., & Schaffer, D. V. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx154 · Cited by 89
Self-citationEmerging applications of gene editing technologies for the development of climate-resilient crops
Chavhan, R. L., Jaybhaye, S. G., Hinge, V. R., Deshmukh, A. S., Shaikh, U. S., Jadhav, P. K., Kadam, U. S., & Hong, J. C. (2025) · Frontiers in Genome Editing · doi:10.3389/fgeed.2025.1524767 · Cited by 55
“Alternatively, when a donor template similar to the target DNA is available, genes can be integrated or corrected via the homology-directed repair (HDR) pathway (Gaj et al., 2016).”
Focused Update on AAV-Based Gene Therapy Clinical Trials for Inherited Retinal Degeneration
Fuller-Carter, P. I., Basiri, H., Harvey, A. R., & Carvalho, L. S. (2020) · BioDrugs · doi:10.1007/s40259-020-00453-8 · Cited by 41
Metadata
- Authors
- Gaj, T., Sirk, S. J., Shui, S.-L., & Liu, J.
- Venue
- Cold Spring Harbor Perspectives in Biology
- Publisher
- Cold Spring Harbor Laboratory
- Published
- 2016-10-06
- Volume
- 8
- Issue
- 12
- Pages
- a023754
- ISSN
- 1943-0264
- Cited by
- 381
- References
- 37
- Language
- en
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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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