ArticleProceedings of the National Academy of Sciences of the United States of America2005
Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 31 citations in OpenAlex.
- Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival.Molecular therapy. Nucleic acids · 2025Article
- Review
- Adeno-Associated Virus and Hematopoietic Stem Cells: The Potential of Adeno-Associated Virus Hematopoietic Stem Cells in Genetic Medicines.Human gene therapy · 2020Review
- Efficient Nuclease-free HR by Clade F AAV Requires High MOIs with High Quality Vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Gene Therapy Approaches to Functional Cure and Protection of Hematopoietic Potential in HIV Infection.Pharmaceutics · 2019Review
- Stem cell-derived clade F AAVs mediate high-efficiency homologous recombination-based genome editing.Proceedings of the National Academy of Sciences of the United States of America · 2018Article
- Adeno-Associated Virus Vectors and Stem Cells: Friends or Foes?Human gene therapy · 2017Review
- Gene transfer properties and structural modeling of human stem cell-derived AAV.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Review
- Article
- Engineering humanized mice for improved hematopoietic reconstitution.Cellular & molecular immunology · 2012Review
- Recombinant AAV2-mediated β-globin expression in human fetal hematopoietic cells from the aborted fetuses with β-thalassemia major.International journal of hematology · 2011Article
- Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2.Human gene therapy · 2010Article
- Genetic modification of ex-vivo expanded stem cells for clinical application.Frontiers in bioscience (Landmark edition) · 2010Review
- The expression of exogenous genes in macrophages: obstacles and opportunities.Methods in molecular biology (Clifton, N.J.) · 2009Article
- Adeno-associated virus-mediated gene transfer.Journal of cellular biochemistry · 2008Article
Corrections and comments
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Authors and funding
8 authors at 3 institutions in 1 country.
Funding
Abstract
A recombinant AAV2 (rAAV2) vector encoding antisense RNA to HIV-1 transactivating region (TAR) was evaluated for transduction of human cord blood CD34+CD38- hematopoietic stem cells (HSC) capable of serial engraftment in nonobese diabetic (NOD)/severe combined immunodeficient (SCID) mice. Results revealed long-term multilineage marking in primary and secondary recipients, and significantly, an enrichment of transduced cells in secondary hosts, indicating efficient transduction of multipotential self-renewing HSC. These results were confirmed by the persistence of rAAV marking of clonogenic progenitors in serial analyses of recipient marrow. Upon HIV-1 challenge, the macrophage progeny of transduced CD34+ cells expressed antisense RNA and exhibited sustained and significant inhibition of virus replication as compared with controls in every donor tested, without selective pressure. This study represents a clear in vivo demonstration of efficient rAAV2 transduction of human HSC.
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Registered trials
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.