A promising genetic approach to the treatment of β-thalassemia Journal Article
| Authors: | May, C.; Sadelain, M. |
| Article Title: | A promising genetic approach to the treatment of β-thalassemia |
| Abstract: | The stable introduction of a functional gene into autologous stem cells is a potentially powerful approach to treat a number of inherited or acquired diseases. One challenge facing this approach is to express adequate levels of the therapeutic transgene in a regulated and sustained fashion, eventually restricting expression to a single lineage developing from the transduced stem cells. Until now, low-level expression, position effects, and transcriptional silencing have hampered the effectiveness of retroviral-mediated gene transfer. In an effort to overcome these obstacles, we have systematically investigated vectors encoding the human β-globin gene linked to selected combinations of proximal and distal genetic regulatory elements. Our results demonstrate that with thoughtful vector design one can successfully express long-term, therapeutic levels of virally encoded human β-globin in the erythroid progeny of hematopoietic stem cells. © 2001, Elsevier Science Inc. |
| Keywords: | promoter region; nonhuman; animals; mice; gene expression; bone marrow; hematopoietic stem cell transplantation; gene transfer; chimera; drug mechanism; gene therapy; beta thalassemia; transgene; beta-thalassemia; short survey; hematopoietic stem cell; gene silencing; lentivirinae; globins; virus vector; gene transfer techniques; enhancer region; beta globin; virus gene; regulator gene; murine leukemia virus; humans; human; priority journal; erythroleukemia cell |
| Journal Title: | Trends in Cardiovascular Medicine |
| Volume: | 11 |
| Issue: | 7 |
| ISSN: | 1050-1738 |
| Publisher: | Elsevier Science, Inc. |
| Date Published: | 2001-10-01 |
| Start Page: | 276 |
| End Page: | 280 |
| Language: | English |
| DOI: | 10.1016/s1050-1738(01)00125-6 |
| PUBMED: | 11709281 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | Export Date: 21 May 2015 -- Source: Scopus |
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