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High-level β-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells
Transplantation of genetically corrected autologous hematopoietic stem cells is an attractive approach for the cure of sickle-cell disease and β-thalassemia. Here, we infected human cord blood cells with a self-inactivating lentiviral vector encoding an anti-sickling β(A-T87Q)-globin transgene and a...
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| Main Authors: | , , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society for Clinical Investigation
2004
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC518665/ https://ncbi.nlm.nih.gov/pubmed/15467834 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI200421838 |
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