Cited by 105Virus-based gene therapy researchCRISPR and Genetic EngineeringRNA Interference and Gene Delivery

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Targeted Genome Modifications Using Integrase-deficient Lentiviral Vectors

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Cornu, Tatjana I, Cathomen, Toni·2007·Molecular Therapy

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Abstract

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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Authors: Cornu, T. I., & Cathomen, T.
Affiliations: Charité – Universitätsmedizin Berlin (DE)
Corresponding author: Cathomen, Toni
ORCID: Cornu, Tatjana I
Cathomen, Toni
Venue: Molecular Therapy
Publisher: Elsevier BV
Published: 2007-12
Volume: 15
Issue: 12
Pages: 2107-2113
ISSN: 1525-0016 (ISSN-L)
DOI: 10.1038/sj.mt.6300345
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: cc-by-nc-nd
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
creativecommons.org/licenses/by-nc-nd/4.0/ (vor, 3233 d)
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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Targeted Genome Modifications Using Integrase-deficient Lentiviral Vectors

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