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Development of Gene Therapy for Thalassemia
Retroviral vector–mediated gene transfer into hematopoietic stem cells provides a potentially curative therapy for severe β-thalassemia. Lentiviral vectors based on human immunodeficiency virus have been developed for this purpose and have been shown to be effective in curing thalassemia in mouse mo...
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| Main Authors: | , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Cold Spring Harbor Laboratory Press
2012
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3543108/ https://ncbi.nlm.nih.gov/pubmed/23125203 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/cshperspect.a011833 |
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