Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
Open accessAbstract
Retroviral vectors derived from lentiviruses such as HIV-1 are promising tools for human gene therapy because they mediate the in vivo delivery and long-term expression of transgenes in nondividing tissues. We describe an HIV vector system in which the virulence genes env, vif, vpr, vpu, and nef have been deleted. This multiply attenuated vector conserved the ability to transduce growth-arrested cells and monocyte-derived macrophages in culture, and could efficiently deliver genes in vivo into adult neurons. These data demonstrate the potential of lentiviral vectors in human gene therapy.
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Most clinical protocols and all commercially approved therapies have relied on viral vectors to deliver CAR transgenes into T cells.
“Further, most clinical protocols, and all the commercially approved therapies, have relied on viral vectors to deliver CAR transgenes into T cells (9-14,24-26).”
From Scalable Manufacturing of CAR T Cells for Cancer Immunotherapy (2021) · cites this paper as [25]
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- Authors
- Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L., & Trono, D.
- Affiliations
- Salk Institute for Biological Studies (US)
- Genesys (United States) (US)
- University of Geneva (CH)
- Corresponding author
- Trono, Didier
- Venue
- Nature Biotechnology
- Published
- 1997-09
- Volume
- 15
- Issue
- 9
- Pages
- 871-875
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- OpenAlex
- W2006519535
- Cited by
- 1,977
- References
- 1
- Citations by year
- 2026: 30 · 2025: 54 · 2024: 52 · 2023: 51 · 2022: 54 · 2021: 76 · 2020: 52 · 2019: 48 · 2018: 61 · 2017: 49 · 2016: 60 · 2015: 65 · 2014: 90 · 2013: 88 · 2012: 85
- FWCI
- 27.08
- Type
- journal article
- Language
- en
- Open access
- green
- infoscience.epfl.ch
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Infoscience (Ecole Polytechnique Fédérale de Lausanne) (submittedVersion)
- Infoscience (Ecole Polytechnique Fédérale de Lausanne) (submittedVersion, cc-by-nc-nd)
- 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 › Immunology and Microbiology › Virology › HIV Research and Treatment
- Keywords
- lentiviral vectors, HIV-1, gene therapy, viral attenuation, nef gene deletion, in vivo gene delivery, adult neurons, monocyte-derived macrophages, transgene expression
- MeSH terms
- Animals, Cells, Cultured, Corpus Striatum, DNA, Viral, Genetic Vectors, Humans, Macrophages, Transduction, Genetic, Transfection, Viral Envelope Proteins, Genetic Therapy, HIV-1, Gene Expression Regulation, Viral, Gene Deletion, Genes, Reporter, Rats
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