The DOI matches its Crossref record; 50 of the 51 references it cites were found in a registry or an index.
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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Cites
A Third-Generation Lentivirus Vector with a Conditional Packaging System
Dull, T., Zufferey, R., Kelly, M., Mandel, R. J., Nguyen, M., Trono, D., & Naldini, L. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8463-8471.1998 · Cited by 3,341
Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery
Zufferey, R., Dull, T., Mandel, R. J., Bukovsky, A., Quiroz, D., Naldini, L., & Trono, D. (1998) · Journal of Virology · doi:10.1128/jvi.72.12.9873-9880.1998 · Cited by 1,815
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
Urnov, F. D., Miller, J. C., Lee, Y.-L., Beausejour, C. M., Rock, J. M., Augustus, S., Jamieson, A. C., Porteus, M. H., Gregory, P. D., & Holmes, M. C. (2005) · Nature · doi:10.1038/nature03556 · Cited by 1,642
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Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning
Aiuti, A., Slavin, S., Aker, M., Ficara, F., Deola, S., Mortellaro, A., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., et al. (2002) · Science · doi:10.1126/science.1070104 · Cited by 1,137
Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy
Hacein‐Bey‐Abina, S., Deist, F. L., Carlier, F., Bouneaud, C., Hue, C., de Villartay, J., Thrasher, A. J., Wulffraat, N. M., Sorensen, R., Dupuis‐Girod, S., et al. (2002) · New England Journal of Medicine · doi:10.1056/nejmoa012616 · Cited by 1,098
An improved zinc-finger nuclease architecture for highly specific genome editing
Miller, J. C., Holmes, M. C., Wang, J., Guschin, D. Y., Lee, Y.-L., Rupniewski, I., Beausejour, C. M., Waite, A. J., Wang, N. S., Kim, K. A., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1319 · Cited by 1,051
Chimeric nucleases stimulate gene targeting in human cells
Porteus, M. H., & Baltimore, D. (2003) · Science · doi:10.1126/science.1078395 · Cited by 741
Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Ponzoni, M., Bartholomae, C. C., Sergi, L. S., Benedicenti, F., Ambrosi, A., Serio, C. D., et al. (2006) · Nature Biotechnology · doi:10.1038/nbt1216 · Cited by 709
Gene targeting using zinc finger nucleases
Porteus, M. H., & Carroll, D. (2005) · Nature Biotechnology · doi:10.1038/nbt1125 · Cited by 641
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Items 101–105 of 105
A systemically-delivered stem cell therapy for dry age related macular degeneration
Pay, S. L. (2017) · IUScholarWorks (Indiana University) · doi:10.7912/c2006q
An improved lentivirus vector for safer transgene integration and protein transduction
Schenkwein, D. (2013) · UEF eRepo (University of Eastern Finland)
Romito, M., Juillerat, A., Kok, Y. L., Hildenbeutel, M., Rhiel, M., Andrieux, G., Geiger, J. P., Rudolph, C., Mussolino, C., Duclert, A., et al. (2020) · doi:10.22541/au.158990011.12602649
Self-citationLentiviral vectors for treatment of haemophilia
Ward, N. J. (2010) · UCL Discovery (University College London)
Development and application of non-integrating lentiviral vectors for gene therapy
Apolonia, L. (2009) · UCL Discovery (University College London)
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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