The DOI matches its Crossref record; 151 of the 152 references it cites were found in a registry or an index.
Integration-deficient Lentiviral Vectors: A Slow Coming of Age
Open accessAbstract
Lentiviral vectors are very efficient at transducing dividing and quiescent cells, which makes them highly useful tools for genetic analysis and gene therapy. Traditionally this efficiency was considered dependent on provirus integration in the host cell genome; however, recent results have challenged this view. So called integration-deficient lentiviral vectors (IDLVs) can be produced through the use of integrase mutations that specifically prevent proviral integration, resulting in the generation of increased levels of circular vector episomes in transduced cells. These lentiviral episomes lack replication signals and are gradually lost by dilution in dividing cells, but are stable in quiescent cells. Compared to integrating lentivectors, IDLVs have a greatly reduced risk of causing insertional mutagenesis and a lower risk of generating replication-competent recombinants (RCRs). IDLVs can mediate transient gene expression in proliferating cells, stable expression in nondividing cells in vitro and in vivo, specific immune responses, RNA interference, homologous recombination (gene repair, knock-in, and knock-out), site-specific recombination, and transposition. IDLVs can be converted into replicating episomes, suggesting that if a clinically applicable system can be developed they would also become highly appropriate for stable transduction of proliferating tissues in therapeutic applications.
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“Thus, NILVs are still the best option for quiescent cells, as critically summarised in one review paper [75].”
From Non-Integrating Lentiviral Vectors in Clinical Applications: A Glance Through (2022) · cites this paper as [75]
In the citing paper
“This approach could help to increase the episomal gene expression, as reported in a previous study where different mutation sites of the integrase plasmid resulted in different gene expression levels [75].”
From Non-Integrating Lentiviral Vectors in Clinical Applications: A Glance Through (2022) · cites this paper as [75]
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Gene therapy development for retinal diseases
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Metadata
- Authors
- Wanisch, K., & Yáñez-Muñoz, R. J.
- Affiliations
- University of London (GB)
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2009-08
- Volume
- 17
- Issue
- 8
- Pages
- 1316-1332
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC2835240
- OpenAlex
- W1976244438
- Cited by
- 222
- References
- 152
- Citations by year
- 2026: 6 · 2025: 7 · 2024: 10 · 2023: 12 · 2022: 12 · 2021: 15 · 2020: 4 · 2019: 8 · 2018: 10 · 2017: 13 · 2016: 14 · 2015: 13 · 2014: 22 · 2013: 19 · 2012: 18
- FWCI
- 1.76
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- UCL Discovery (University College London) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Keywords
- integrase mutations, proviral integration, insertional mutagenesis, quiescent cells, transient expression, stable transduction, homologous recombination, site-specific recombination, transposition, gene therapy
- MeSH terms
- Animals, Genetic Vectors, Humans, Mutation, Transduction, Genetic, Lentivirus, Integrases
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