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Targeted Genome Modifications Using Integrase-deficient Lentiviral Vectors
Open accessAbstract
Gene correction aims at repairing a defective gene directly in the cellular genome, which warrants tissue-specific and sustained expression of the repaired gene through its endogenous promoter. We have developed a novel system based on integrase-deficient lentiviral vectors (IDLVs) that allows us to correct an endogenous mutation using a strategy based on homologous recombination (HR). In a proof-of-concept approach, an IDLV encoding a repair template was co-delivered with an I-SceI nuclease expression vector to rescue a defective enhanced green fluorescent protein (EGFP) gene. Expression of the nuclease created a double-strand break within the target locus, which was crucial for stimulating IDLV-based gene repair. Stable gene correction was realized in up to 12% of the cells, depending on the vector dose, the nuclease expression levels, and the cell type. Genotypic analyses confirmed that gene correction was the result of genuine HR between the target locus and the IDLV repair template. This study presents IDLVs as valuable tools for introducing precise and permanent genetic modifications in human cells.
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“For example, an NILV encoding a repair template was co-transported with an I-SceI nuclease expression vector to rescue a defective enhanced green fluorescent protein (EGFP) gene. Expression of the nuclease created a double-strand break (DSB) within the targeted locus and stimulated stable gene correction at the end of the process [21].”
From Non-Integrating Lentiviral Vectors in Clinical Applications: A Glance Through (2022) · cites this paper as [21]
In the citing paper
“NILVs can also be co-transduced into cells with zinc finger nucleases, which facilitate recombination of DNA encoded in the vector with a specific site in the host DNA [89].”
From Clinical use of lentiviral vectors (2018) · cites this paper as [89]
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Clinical use of lentiviral vectors
Milone, M. C., & O’Doherty, U. (2018) · Leukemia · doi:10.1038/s41375-018-0106-0 · Cited by 930
“NILVs can also be co-transduced into cells with zinc finger nucleases, which facilitate recombination of DNA encoded in the vector with a specific site in the host DNA [89].”
BackgroundAn unbiased genome-wide analysis of zinc-finger nuclease specificity
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Metadata
- Authors
- Cornu, T. I., & Cathomen, T.
- Affiliations
- Charité – Universitätsmedizin Berlin (DE)
- Corresponding author
- Cathomen, Toni
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2007-12
- Volume
- 15
- Issue
- 12
- Pages
- 2107-2113
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W1974572528
- Cited by
- 105
- References
- 51
- Citations by year
- 2026: 1 · 2025: 3 · 2024: 2 · 2022: 2 · 2021: 3 · 2020: 4 · 2019: 1 · 2018: 2 · 2017: 7 · 2016: 4 · 2015: 6 · 2014: 10 · 2013: 5 · 2012: 11
- FWCI
- 5.18
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- FreiDok plus (Universitätsbibliothek Freiburg) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- 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
- Keywords
- gene correction, homologous recombination, DNA double-strand breaks, targeted genome modification, human cells, gene therapy
- MeSH terms
- Base Sequence, Genetic Vectors, Genotype, Transduction, Genetic, Gene Expression, Lentivirus, Polymerase Chain Reaction, Genome, DNA Primers
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