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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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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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