ArticleMolecular therapy : the journal of the American Society of Gene Therapy2008
A large U3 deletion causes increased in vivo expression from a nonintegrating lentiviral vector.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers.
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Who cites it
62 citing papers in PubMed, 96 citations in OpenAlex.
- CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.Molecular therapy. Nucleic acids · 2026Review
- Analysis of Hepatic Lentiviral Vector Transduction: Implications for Preclinical Studies and Clinical Gene Therapy Protocols.Viruses · 2025Article
- Engineering single-cycle MeV vector for CRISPR-Cas9 gene editing.Molecular therapy. Methods & clinical development · 2024Article
- Analysis of hepatic lentiviral vector transduction; implications for preclinical studies and clinical gene therapy protocols.bioRxiv : the preprint server for biology · 2024Article
- The potential of gene delivery for the treatment of traumatic brain injury.Journal of neuroinflammation · 2024Review
- Advances of Genome Editing with CRISPR/Cas9 in Neurodegeneration: The Right Path towards Therapy.Biomedicines · 2023Review
- HIV Preintegration Transcription and Host Antagonism.Current HIV research · 2023Article
- Autologous skin-derived neural precursor cell therapy reverses canine Alzheimer dementia-like syndrome in a proof of concept veterinary trial.Stem cell research & therapy · 2022Article
- Review
- Therapeutic modulation of gene expression in the disease state: Treatment strategies and approaches for the development of next-generation of the epigenetic drugs.Frontiers in bioengineering and biotechnology · 2022Review
- Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization.Molecular therapy. Methods & clinical development · 2021Article
- Lentiviral Vectors for Delivery of Gene-Editing Systems Based on CRISPR/Cas: Current State and Perspectives.Viruses · 2021Review
- Tax Induces the Recruitment of NF-κB to Unintegrated HIV-1 DNA To Rescue Viral Gene Expression and Replication.Journal of virology · 2021Article
- Review
- Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases.Frontiers in molecular neuroscience · 2020Article
- Gene Delivery to Human Limbal Stem Cells Using Viral Vectors.Human gene therapy · 2019Article
- Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models.Journal of visualized experiments : JoVE · 2019Article
- Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy for Precision Therapy in PD.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization.Gene therapy · 2018Article
- Improving the Transduction of Bone Marrow-Derived Cells with an Integrase-Defective Lentiviral Vector.Human gene therapy methods · 2018Article
2 more citing papers are in PubMed but not listed here.
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Authors and funding
8 authors at 1 institution in 1 country.
Funding
Abstract
The feasibility of using nonintegrating lentiviral vectors has been demonstrated by recent studies showing their ability to maintain transgene expression both in vitro and in vivo. Furthermore, human immunodeficiency virus-1 (HIV-1) vectors packaged with a mutated integrase were able to correct retinal disease in a mouse model. Interestingly, these results differ from earlier studies in which first-generation nonintegrating lentiviral vectors yielded insignificant levels of transduction. However, to date, a rigorous characterization of transgene expression from the currently used self-inactivating (SIN) nonintegrating lentiviral vectors has not been published. In this study, we characterize transgene expression from SIN nonintegrating lentiviral vectors. Overall, we found that nonintegrating vectors express transgenes at a significantly lower level than their integrating counterparts. Expression from nonintegrating vectors was improved upon introducing a longer deletion in the vector's U3 region. A unique shuttle-vector assay indicated that the relative abundance of the different episomal forms was not altered by the longer U3 deletion. Interestingly, the longer U3 deletion did not enhance expression in the corpus callosum of the rat brain, suggesting that the extent of silencing of episomal transcription is influenced by tissue-specific factors. Finally, and for the first time, episomal expression in the mouse liver was potent and sustained.
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