The DOI matches its Crossref record; 52 of the 52 references it cites were found in a registry or an index.
Advances in therapeutic CRISPR/Cas9 genome editing
Open accessAbstract
Targeted nucleases are widely used as tools for genome editing. Two years ago the clustered regularly interspaced short palindromic repeat (CRISPR)-associated Cas9 nuclease was used for the first time, and since then has largely revolutionized the field. The tremendous success of the CRISPR/Cas9 genome editing tool is powered by the ease design principle of the guide RNA that targets Cas9 to the desired DNA locus, and by the high specificity and efficiency of CRISPR/Cas9-generated DNA breaks. Several studies recently used CRISPR/Cas9 to successfully modulate disease-causing alleles in vivo in animal models and ex vivo in somatic and induced pluripotent stem cells, raising hope for therapeutic genome editing in the clinics. In this review, we will summarize and discuss such preclinical CRISPR/Cas9 gene therapy reports.
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“CRISPR/Cas9 can change the cancer-related genes and improve the ability of immune cells to recognize and get rid of cancer cells, opening up new avenues for cancer immunotherapy.[6]”
From A Review of Research on CRISPR/Cas9 Gene Editing Technology in Disease Treatment (2024) · cites this paper as [6]
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Cites
Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors
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A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
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Multiplex Genome Engineering Using CRISPR/Cas Systems
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RNA-Guided Human Genome Engineering via Cas9
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The new frontier of genome engineering with CRISPR-Cas9
Doudna, J. A., & Charpentier, E. (2014) · Science · doi:10.1126/science.1258096 · Cited by 7,062
Sato, T., Stange, D. E., Ferrante, M., Vries, R. G. J., van Es, J. H., van den Brink, S., van Houdt, W. J., Pronk, A., van Gorp, J., Siersema, P. D., et al. (2011) · Gastroenterology · doi:10.1053/j.gastro.2011.07.050 · Cited by 3,753
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Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
Ran, F. A., Hsu, P. D., Lin, C.-Y., Gootenberg, J. S., Konermann, S., Trevino, A. E., Scott, D. A., Inoue, A., Matoba, S., Zhang, Y., et al. (2013) · Cell · doi:10.1016/j.cell.2013.08.021 · Cited by 3,299
High-Frequency off-Target Mutagenesis Induced by CRISPR-Cas Nucleases in Human Cells
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In vivo genome editing using Staphylococcus aureus Cas9
Ran, F. A., Cong, L., Yan, W. X., Scott, D. A., Gootenberg, J. S., Kriz, A. J., Zetsche, B., Shalem, O., Wu, X., Makarova, K. S., et al. (2015) · Nature · doi:10.1038/nature14299 · Cited by 2,687
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CRISPR-Cas: biology, mechanisms and relevance
Hille, F., & Charpentier, E. (2016) · Philosophical Transactions of the Royal Society B: Biological Sciences · doi:10.1098/rstb.2015.0496 · Cited by 442
Gadhave, D. G., Sugandhi, V. V., Jha, S. K., Nangare, S., Gupta, G., Singh, S. K., Dua, K., Cho, H., Hansbro, P. M., & Paudel, K. R. (2024) · Ageing Research Reviews · doi:10.1016/j.arr.2024.102357 · Cited by 420
Engineering the delivery system for CRISPR-based genome editing
Glass, Z., Lee, M., Li, Y., & Xu, Q. (2018) · Trends in biotechnology · doi:10.1016/j.tibtech.2017.11.006 · Cited by 345
Huntingtin Lowering Strategies for Disease Modification in Huntington’s Disease
Tabrizi, S. J., Ghosh, R., & Leavitt, B. R. (2019) · Neuron · doi:10.1016/j.neuron.2019.01.039 · Cited by 336
Therapies targeting DNA and RNA in Huntington's disease
Wild, E. J., & Tabrizi, S. J. (2017) · The Lancet Neurology · doi:10.1016/s1474-4422(17)30280-6 · Cited by 307
Metadata
- Authors
- Savić, N., & Schwank, G.
- Affiliations
- ETH Zurich (CH)
- Corresponding author
- Schwank, Gerald
- Venue
- Translational Research
- Publisher
- Elsevier BV
- Published
- 2016-02
- Volume
- 168
- Pages
- 15-21
- ISSN
- 1878-1810 (ISSN-L)
- 1931-5244
- OpenAlex
- W1804459219
- Cited by
- 257
- References
- 52
- Citations by year
- 2026: 7 · 2025: 20 · 2024: 29 · 2023: 21 · 2022: 26 · 2021: 25 · 2020: 31 · 2019: 25 · 2018: 26 · 2017: 22 · 2016: 25
- FWCI
- 2.36
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Translational research (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Repository for Publications and Research Data (ETH Zurich) (submittedVersion, cc-by-nc-nd)
- ETH Zürich Research Collection
- Funders
- Swiss National Science Foundation (31003A_160230)
- Human Frontiers in Science Program (LT000422/2012)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Keywords
- CRISPR/Cas9, genome editing, gene therapy, guide RNA, somatic cells, induced pluripotent stem cells, animal models
- MeSH terms
- CRISPR-Associated Protein 9, Animals, Bacterial Proteins, Endonucleases, Gene Expression Regulation, Genetic Therapy, Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR-Cas Systems
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