ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
High-efficiency transduction of rhesus hematopoietic repopulating cells by a modified HIV1-based lentiviral vector.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.
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Who cites it
28 citing papers in PubMed, 36 citations in OpenAlex.
- Efficient transduction of human hematopoietic repopulating cells with a chimeric HIV1-based vector including SIV capsid.Experimental hematology · 2013Trial
- 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
- Hematopoietic stem cell therapy with gene modification to treat sickle cell disease.Stem cells translational medicine · 2025Review
- Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model.Nature communications · 2023Article
- A Comprehensive Atlas of Immunological Differences Between Humans, Mice, and Non-Human Primates.Frontiers in immunology · 2022Article
- Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease.Molecular therapy. Methods & clinical development · 2021Article
- Preclinical evaluation for engraftment of CD34Cell reports. Medicine · 2021Article
- NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease.Blood advances · 2020Article
- Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders.Nature communications · 2019Article
- Busulfan Combined with Immunosuppression Allows Efficient Engraftment of Gene-Modified Cells in a Rhesus Macaque Model.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Fetal hemoglobin and F-cell variance in mobilized CD34Experimental hematology · 2019Article
- High-Efficiency Lentiviral Transduction of Human CD34Molecular therapy. Methods & clinical development · 2019Article
- Chimeric Antigen Receptor T Cell-Mediated Neurotoxicity in Nonhuman Primates.Cancer discovery · 2018Article
- Geographic clonal tracking in macaques provides insights into HSPC migration and differentiation.The Journal of experimental medicine · 2018Article
- Building a Safer and Faster CAR: Seatbelts, Airbags, and CRISPR.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2018Review
- 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
- RNA Trans-Splicing Targeting Endogenous β-Globin Pre-Messenger RNA in Human Erythroid Cells.Human gene therapy methods · 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
- Kinetics of lentiviral vector transduction in human CD34(+) cells.Experimental hematology · 2016Article
Corrections and comments
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Authors and funding
14 authors at 5 institutions in 1 country.
Funding
Abstract
Human immunodeficiency virus type 1 (HIV1) vectors poorly transduce rhesus hematopoietic cells due to species-specific restriction factors, including the tripartite motif-containing 5 isoformα (TRIM5α) which targets the HIV1 capsid. We previously developed a chimeric HIV1 (χHIV) vector system wherein the vector genome is packaged with the simian immunodeficiency virus (SIV) capsid for efficient transduction of both rhesus and human CD34(+) cells. To evaluate whether χHIV vectors could efficiently transduce rhesus hematopoietic repopulating cells, we performed a competitive repopulation assay in rhesus macaques, in which half of the CD34(+) cells were transduced with standard SIV vectors and the other half with χHIV vectors. As compared with SIV vectors, χHIV vectors achieved higher vector integration, and the transgene expression rates were two- to threefold higher in granulocytes and red blood cells and equivalent in lymphocytes and platelets for 2 years. A recipient of χHIV vector-only transduced cells reached up to 40% of transgene expression rates in granulocytes and lymphocytes and 20% in red blood cells. Similar to HIV1 and SIV vectors, χHIV vector frequently integrated into gene regions, especially into introns. In summary, our χHIV vector demonstrated efficient transduction for rhesus long-term repopulating cells, comparable with SIV vectors. This χHIV vector should allow preclinical testing of HIV1-based therapeutic vectors in large animal models.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.