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A promising genetic approach to the treatment of beta-thalassemia.
- Source :
-
Trends in cardiovascular medicine [Trends Cardiovasc Med] 2001 Oct; Vol. 11 (7), pp. 276-80. - Publication Year :
- 2001
-
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 beta-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 beta-globin in the erythroid progeny of hematopoietic stem cells.
Details
- Language :
- English
- ISSN :
- 1050-1738
- Volume :
- 11
- Issue :
- 7
- Database :
- MEDLINE
- Journal :
- Trends in cardiovascular medicine
- Publication Type :
- Academic Journal
- Accession number :
- 11709281
- Full Text :
- https://doi.org/10.1016/s1050-1738(01)00125-6