ArticleProceedings of the National Academy of Sciences of the United States of America2000
Long-term expression of gamma-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human gamma-globin gene fused to the ankyrin-1 promoter.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2000. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 46 citations in OpenAlex.
- The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges.Therapeutic advances in hematology · 2016Review
- Gene therapy for hemoglobinopathies: progress and challenges.Translational research : the journal of laboratory and clinical medicine · 2013Review
- Development of gene therapy for thalassemia.Cold Spring Harbor perspectives in medicine · 2012Review
- Article
- Gene therapy in thalassemia and hemoglobinopathies.Mediterranean journal of hematology and infectious diseases · 2009Article
- Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Absence of retroviral vector-mediated transformation of gene-modified T cells after long-term engraftment in mice.Gene therapy · 2008Article
- Effective gene therapy of mice with congenital erythropoietic porphyria is facilitated by a survival advantage of corrected erythroid cells.American journal of human genetics · 2008Article
- Modification of globin gene expression by RNA targeting strategies.Experimental hematology · 2007Article
- Erythroid-specific expression of beta-globin by the sleeping beauty transposon for Sickle cell disease.Biochemistry · 2007Article
- Altered regulation of beta-like globin genes by a redesigned erythroid transcription factor.Experimental hematology · 2007Article
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy for patients with hemoglobin disorders has been hampered by the inability of retrovirus vectors to transfer globin genes and their cis-acting regulatory sequences into hematopoietic stem cells without rearrangement. In addition, the expression from intact globin gene vectors has been variable in red blood cells due to position effects and retrovirus silencing. We hypothesized that by substituting the globin gene promoter for the promoter of another gene expressed in red blood cells, we could generate stable retrovirus vectors that would express globin at sufficient levels to treat hemoglobinopathies. Recently, we have shown that the human ankyrin (Ank) gene promoter directs position-independent, copy number-dependent expression of a linked gamma-globin gene in transgenic mice. We inserted the Ank/(A)gamma-globin gene into retrovirus vectors that could transfer one or two copies of the Ank/(A)gamma-globin gene to target cells. Both vectors were stable, transferring only intact proviral sequences into primary mouse hematopoietic stem cells. Expression of Ank/(A)gamma-globin mRNA in mature red blood cells was 3% (single copy) and 8% (double copy) of the level of mouse alpha-globin mRNA. We conclude that these novel retrovirus vectors may be valuable for treating a variety of red cell disorders by gene replacement therapy including severe beta-thalassemia if the level of expression can be further increased.
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