Current and future delivery systems for engineered nucleases: ZFN, TALEN and RGEN
Abstract
Gene therapy by engineered nucleases is a genetic intervention being investigated for curing the hereditary disorders by targeting selected genes with specific nucleotides for establishment, suppression, abolishment of a function or correction of mutation. Here, we review the fast developing technology of targeted genome engineering using site specific programmable nucleases zinc finger nucleases (ZFNs), transcription activator like nucleases (TALENs) and cluster regulatory interspaced short palindromic repeat/CRISPR associated proteins (CRISPR/Cas) based RNA-guided DNA endonucleases (RGENs) and their different characteristics including pros and cons of genome modifications by these nucleases. We have further discussed different types of delivery methods to induce gene editing, novel development in genetic engineering other than nucleases and future prospects.
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The availability of genomic sequences has enabled CHO cell engineering with unprecedented precision using engineered nucleases including ZFNs, TALENs and CRISPR.
“The availability of genomic sequences has enabled CHO cell engineering with unprecedented precision using engineered nucleases, including zinc finger nucleases (ZFNs) and transcription activator like nucleases (TALENs) and the clustered regularly interspaced short palindromic repeats (CRISPR) system [71–73,75,76].”
From Glycoengineering Chinese hamster ovary cells: a short history (2021) · cites this paper as [76]
Cites
Genome-scale CRISPR-Cas9 knockout screening in human cells
Shalem, O., Sanjana, N. E., Hartenian, E., Shi, X., Scott, D. A., Mikkelsen, T. S., Heckl, D., Ebert, B. L., Root, D. E., Doench, J. G., et al. (2014) · Science · doi:10.1126/science.1247005 · Cited by 5,234 · see citing works
Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
Qi, L. S., Larson, M. H., Gilbert, L. A., Doudna, J. A., Weissman, J. S., Arkin, A. P., & Lim, W. A. (2013) · Cell · doi:10.1016/j.cell.2013.02.022 · Cited by 5,016 · see citing works
Efficient genome editing in zebrafish using a CRISPR-Cas system
Hwang, W. Y., Fu, Y., Reyon, D., Maeder, M. L., Tsai, S. Q., Sander, J. D., Peterson, R. T., Yeh, J.-R. J., & Joung, J. K. (2013) · Nature Biotechnology · doi:10.1038/nbt.2501 · Cited by 2,937 · see citing works
RNA-guided editing of bacterial genomes using CRISPR-Cas systems
Jiang, W., Bikard, D., Cox, D., Zhang, F., & Marraffini, L. A. (2013) · Nature Biotechnology · doi:10.1038/nbt.2508 · Cited by 2,429 · see citing works
RNA-guided genetic silencing systems in bacteria and archaea
Wiedenheft, B., Sternberg, S. H., & Doudna, J. A. (2012) · Nature · doi:10.1038/nature10886 · Cited by 1,851 · see citing works
TALENs: A widely applicable technology for targeted genome editing
Joung, J. K., & Sander, J. D. (2013) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm3486 · Cited by 1,650 · see citing works
Schwank, G., Koo, B.-K., Sasselli, V., Dekkers, J. F., Heo, I., Demircan, T., Sasaki, N., Boymans, S., Cuppen, E., van der Ent, C. K., et al. (2013) · Cell Stem Cell · doi:10.1016/j.stem.2013.11.002 · Cited by 1,424 · see citing works
Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA
Ramakrishna, S., Kwaku Dad, A.-B., Beloor, J., Gopalappa, R., Lee, S.-K., & Kim, H. (2014) · Genome Research · doi:10.1101/gr.171264.113 · Cited by 655 · see citing works
In 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 · see citing works
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 · see citing works
Li, L., Krymskaya, L., Wang, J., Henley, J., Rao, A., Cao, L.-F., Tran, C.-A., Torres-Coronado, M., Gardner, A., Gonzalez, N., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.65 · Cited by 181 · see citing works
Maier, D. A., Brennan, A. L., Jiang, S., Binder-Scholl, G. K., Lee, G., Plesa, G., Zheng, Z., Cotte, J., Carpenito, C., Wood, T., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.172 · Cited by 126 · see citing works
Targeted Genome Modifications Using Integrase-deficient Lentiviral Vectors
Cornu, T. I., & Cathomen, T. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300345 · Cited by 105 · see citing works
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Lin, Y., Wu, J., Gu, W., Huang, Y., Tong, Z., Huang, L., & Tan, J. (2018) · Advanced Science · doi:10.1002/advs.201700611 · Cited by 625 · see citing works
Wang, H.-X., Li, M., Lee, C. M., Chakraborty, S., Kim, H.-W., Bao, G., & Leong, K. W. (2017) · Chemical Reviews · doi:10.1021/acs.chemrev.6b00799 · Cited by 560 · see citing works
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- Authors
- Ain, Q. U., Chung, J. Y., & Kim, Y.-H.
- Affiliations
- Hanyang University (KR)
- Corresponding author
- Kim, Yong-Hee
- Venue
- Journal of Controlled Release
- Publisher
- Elsevier BV
- Published
- 2015-05
- Volume
- 205
- Pages
- 120-127
- ISSN
- 0168-3659 (ISSN-L)
- OpenAlex
- W2042983080
- Cited by
- 130
- References
- 13
- Citations by year
- 2026: 5 · 2025: 11 · 2024: 9 · 2023: 5 · 2022: 12 · 2021: 13 · 2020: 16 · 2019: 7 · 2018: 13 · 2017: 10 · 2016: 17 · 2015: 12
- FWCI
- 1.22
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Journal of Controlled Release (publishedVersion)
- PubMed (publishedVersion)
- The Royal Society of Chemistry’s Journals, Books and Databases (The Royal Society of Chemistry) (submittedVersion)
- Funders
- National Research Foundation of Korea (2014049587)
- Brain Korea 21 plus program (22A20130011095)
- Korean Health Technology R&D project (HI13C-1938-010014)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- engineered nucleases, gene delivery systems, zinc finger nucleases, TALEN, CRISPR-Cas, RNA-guided DNA endonuclease, genome editing, gene therapy, genetic disorders
- MeSH terms
- Animals, Gene Expression Regulation, Genetic Vectors, Humans, Ribonucleases, Viruses, Protein Engineering, Genetic Therapy, Zinc Fingers, Gene Transfer Techniques, Targeted Gene Repair, Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR-Cas Systems, CRISPR-Associated Proteins
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