A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity
Open accessAbstract
Sequence-specific nucleases represent valuable tools for precision genome engineering. Traditionally, zinc-finger nucleases (ZFNs) and meganucleases have been used to specifically edit complex genomes. Recently, the DNA binding domains of transcription activator-like effectors (TALEs) from the bacterial pathogen Xanthomonas have been harnessed to direct nuclease domains to desired genomic loci. In this study, we tested a panel of truncation variants based on the TALE protein AvrBs4 to identify TALE nucleases (TALENs) with high DNA cleavage activity. The most favorable parameters for efficient DNA cleavage were determined in vitro and in cellular reporter assays. TALENs were designed to disrupt an EGFP marker gene and the human loci CCR5 and IL2RG. Gene editing was achieved in up to 45% of transfected cells. A side-by-side comparison with ZFNs showed similar gene disruption activities by TALENs but significantly reduced nuclease-associated cytotoxicities. Moreover, the CCR5-specific TALEN revealed only minimal off-target activity at the CCR2 locus as compared to the corresponding ZFN, suggesting that the TALEN platform enables the design of nucleases with single-nucleotide specificity. The combination of high nuclease activity with reduced cytotoxicity and the simple design process marks TALENs as a key technology platform for targeted modifications of complex genomes.
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The only attempt using the TALEN system for HIV-1 gene therapy was reported in 2011.
“So far, the only attempt using the TALEN system for HIV-1 gene therapy was reported in 2011 [81].”
From Newer Gene Editing Technologies toward HIV Gene Therapy (2013) · cites this paper as [81]
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- Authors
- Mussolino, C., Morbitzer, R., Lütge, F., Dannemann, N., Lahaye, T., & Cathomen, T.
- Affiliations
- Medizinische Hochschule Hannover (DE)
- Corresponding author
- Cathomen, Toni
- Venue
- Nucleic Acids Research
- Publisher
- Oxford University Press (OUP)
- Published
- 2011-08-03
- Volume
- 39
- Issue
- 21
- Pages
- 9283-9293
- ISSN
- 0305-1048 (ISSN-L)
- 1362-4962
- PMC
- PMC3241638
- OpenAlex
- W2138231299
- Cited by
- 714
- References
- 52
- Citations by year
- 2026: 7 · 2025: 12 · 2024: 15 · 2023: 19 · 2022: 23 · 2021: 33 · 2020: 35 · 2019: 34 · 2018: 40 · 2017: 62 · 2016: 66 · 2015: 77 · 2014: 111 · 2013: 111 · 2012: 56
- FWCI
- 37.56
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Nucleic Acids Research (publishedVersion, cc-by-nc)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- FreiDok plus (Universitätsbibliothek Freiburg) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- TALE nucleases, transcription activator-like effectors, zinc finger nucleases, genome editing, CCR5, IL2RG, off-target effects, DNA cleavage activity, Xanthomonas
- MeSH terms
- Transcription Activator-Like Effectors, Bacterial Proteins, Deoxyribonucleases, DNA-Binding Proteins, Genetic Engineering, Humans, Protein Engineering, Trans-Activators, Genome, Human, Protein Structure, Tertiary, Gene Targeting, DNA Cleavage, HEK293 Cells
About these checks
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.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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