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Lenti in red: progress in gene therapy for human hemoglobinopathies
Hemoglobinopathies are caused by abnormal structure or synthesis of hemoglobin chains and represent serious monogenic disorders. A new study demonstrates that lentiviral vectors can express clinically relevant levels of human transgenic β-globin in red cells of xenografted mice. While some safety co...
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| Autors principals: | , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society for Clinical Investigation
2004
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC518672/ https://ncbi.nlm.nih.gov/pubmed/15467825 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI200423132 |
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