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Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
Published on: March 29, 2019
Making lentiviral vectors more powerful and universal.
1Sidney Kimmel Comprehensive Cancer Center, The Johns Hopkins University School of Medicine, 1650 Orleans Street, Baltimore, MD 21231, USA.
Discovery Medicine
|August 14, 2010
Summary
Lentiviral vectors (LV) enable efficient gene transfer into primary mammalian cells, including non-dividing cells like hematopoietic stem cells and T cells. Their self-inactivating modification ensures safety without reducing viral titers, offering advantages over older retroviral vectors (RV).
Area of Science:
- Molecular Biology
- Gene Therapy
- Virology
Background:
- Recombinant retroviral vectors are essential for stable gene delivery in primary mammalian cells.
- Lentiviral vectors (LV) and onco-retroviral vectors (RV) are key tools in this field.
- Previous work established LV for simultaneous or independent gene expression and efficient gene transfer into human T lymphocytes.
Purpose of the Study:
- To highlight the advantages of lentiviral vectors (LV) over onco-retroviral vectors (RV) for gene delivery.
- To emphasize the utility of LV for transducing both dividing and non-dividing primary cells.
- To discuss the safety and efficacy of self-inactivating (SIN) LV modifications.
Main Methods:
- Construction and utilization of lentiviral vectors (LV) for gene expression.
- Gene transfer experiments into human T lymphocytes.
- Comparison of LV with onco-retroviral vectors (RV) regarding transduction efficiency and safety features.
Main Results:
- LV can efficiently transduce both dividing and non-dividing primary mammalian cells, including hematopoietic stem cells and T cells.
- LV accommodate diverse promoters for cell-specific or ubiquitous gene expression.
- Self-inactivating (SIN) LV modifications enhance safety by disabling viral promoters post-integration without reducing viral titers, unlike SIN RVs.
Conclusions:
- Lentiviral vectors offer significant advantages over traditional retroviral vectors for stable gene delivery.
- The ability to transduce non-dividing cells and the safety features of SIN-LV make them a superior choice for gene therapy applications.
- LV provide a versatile and effective platform for genetic modification of primary cells.
