ArticleThe Journal of clinical investigation2003
Hematopoietic stem cell gene therapy: selecting only the best.
Article in The Journal of clinical investigation, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
8 citing papers in PubMed.
- Immune tolerance induced by platelet-targeted factor VIII gene therapy in hemophilia A mice is CD4 T cell mediated.Journal of thrombosis and haemostasis : JTH · 2017Article
- Development of autologous blood cell therapies.Experimental hematology · 2016Review
- Transgene expression in various organs post BM-HSC transplantation.Stem cell research · 2014Article
- Comparison of transduction efficiency among various lentiviruses containing GFP reporter in bone marrow hematopoietic stem cell transplantation.Experimental hematology · 2013Article
- Lentiviral Based Gene Transduction and Promoter Studies in Human Hematopoietic Stem Cells (hHSCs).Journal of stem cells & regenerative medicine · 2011Article
- Characterization of retroviral and lentiviral vectors pseudotyped with xenotropic murine leukemia virus-related virus envelope glycoprotein.Human gene therapy · 2010Article
- DNA repair proteins as molecular targets for cancer therapeutics.Anti-cancer agents in medicinal chemistry · 2008Review
- Live and let die: in vivo selection of gene-modified hematopoietic stem cells via MGMT-mediated chemoprotection.DNA repair · 2007Review
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1 author.
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Abstract
Hematopoietic stem cell (HSC) gene therapy can potentially cure a variety of human hematopoietic diseases, such as sickle cell disease. Selection and expansion of gene-corrected HSCs has now been accomplished for the first time using HSC from large animals - dogs and humans - with a novel drug-resistance gene, MGMT, which is not expressed in normal HSCs (see the related articles beginning on pages 1561 and 1581). Highly efficient lentiviral transfer and expression of MGMT into relatively few HSCs led to repopulation of most of the hematopoietic compartment with gene-corrected cells following suitable drug treatment. This selection system may be useful in human clinical trials to permit gene therapy in autologous and allogeneic bone marrow transplantation settings.
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