ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
TRIM5α variations influence transduction efficiency with lentiviral vectors in both human and rhesus CD34(+) cells in vitro and in vivo.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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Who cites it
21 citing papers in PubMed, 28 citations in OpenAlex.
- Targeted in vivo delivery of genetic medicines utilizing an engineered lentiviral vector platform results in CAR T and NK cell generation.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction.Cells · 2024Review
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- Genome editing in large animal models.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease.Molecular therapy. Methods & clinical development · 2021Article
- Knockdown of TRIM5α or TRIM11 increases lentiviral vector transduction efficiency of human Muller cells.Experimental eye research · 2021Article
- Antiviral immunity and nucleic acid sensing in haematopoietic stem cell gene engineering.Gene therapy · 2021Review
- BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity.The Journal of clinical investigation · 2020Article
- Hematopoietic Stem/Progenitor Cells and the Pathogenesis of HIV/AIDS.Frontiers in cellular and infection microbiology · 2020Review
- Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders.Nature communications · 2019Article
- X-chromosome-linked miR548am-5p is a key regulator of sex disparity in the susceptibility to mitochondria-mediated apoptosis.Cell death & disease · 2019Article
- Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells.Cell stem cell · 2018Article
- Chimeric Antigen Receptor T Cell-Mediated Neurotoxicity in Nonhuman Primates.Cancer discovery · 2018Article
- Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients.Human gene therapy. Clinical development · 2017Article
- Efficient Transduction of Human and Rhesus Macaque Primary T Cells by a Modified Human Immunodeficiency Virus Type 1-Based Lentiviral Vector.Human gene therapy · 2017Article
- Article
- Kinetics of lentiviral vector transduction in human CD34(+) cells.Experimental hematology · 2016Article
- Cyclosporin a and rapamycin relieve distinct lentiviral restriction blocks in hematopoietic stem and progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Pigtailed macaques as a model to study long-term safety of lentivirus vector-mediated gene therapy for hemoglobinopathies.Molecular therapy. Methods & clinical development · 2014Article
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 1 country.
Funding
Abstract
Human immunodeficiency virus type 1 (HIV-1) vectors can transduce human hematopoietic stem cells (HSC), but transduction efficiency varies among individuals. The innate immune factor tripartite motif-containing protein 5α (TRIM5α) plays an important role for restriction of retroviral infection. In this study, we examined whether TRIM5α could account for variations in transduction efficiency using both an established rhesus gene therapy model and human CD34(+) cell culture. Evaluation of TRIM5α genotypes (Mamu-1, -2, -3, -4, -5, and TrimCyp) in 16 rhesus macaques that were transplanted with transduced CD34(+) cells showed a significant correlation between TRIM5α Mamu-4 and high gene marking in both lymphocytes and granulocytes 6 months after transplantation. Since significant human TRIM5α coding polymorphisms were not known, we evaluated TRIM5α expression levels in human CD34(+) cells from 14 donors. Three days after HIV-1 vector transduction, measured transduction efficiency varied significantly among donors and was negatively correlated with TRIM5α expression levels. In summary, transduction efficiency in both rhesus and human CD34(+) cells was influenced by TRIM5α variations (genotypes and expression levels). Our findings are important for both understanding and mitigating the variability of transduction efficiency for rhesus and human CD34(+) cells.
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