Cited by 257CRISPR and Genetic EngineeringVirus-based gene therapy researchRNA regulation and disease

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Advances in therapeutic CRISPR/Cas9 genome editing

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Savić, Nataša, Schwank, Gerald·2016·Translational Research

DOI 10.1016/j.trsl.2015.09.008

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Abstract

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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Authors: Savić, N., & Schwank, G.
Affiliations: ETH Zurich (CH)
Corresponding author: Schwank, Gerald
ORCID: Savić, Nataša
Schwank, Gerald
Venue: Translational Research
Publisher: Elsevier BV
Published: 2016-02
Volume: 168
Pages: 15-21
ISSN: 1878-1810 (ISSN-L)
1931-5244
DOI: 10.1016/j.trsl.2015.09.008
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: cc-by-nc-nd
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
creativecommons.org/licenses/by-nc-nd/4.0/ (vor)
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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