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Correction of murine hemophilia A following nonmyeloablative transplantation of hematopoietic stem cells engineered to encode an enhanced human factor VIII variant using a safety-augmented retroviral vector
Insertional mutagenesis by retroviral vectors is a major impediment to the clinical application of hematopoietic stem cell gene transfer for the treatment of hematologic disorders. We recently developed an insulated self-inactivating gammaretroviral vector, RMSinOFB, which uses a novel enhancer-bloc...
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| Main Authors: | , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Hematology
2009
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| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2713478/ https://ncbi.nlm.nih.gov/pubmed/19470695 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2009-01-199653 |
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