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Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
Hematopoietic stem cell (HSC) gene therapy is being evaluated for hemoglobin disorders including sickle cell disease (SCD). Therapeutic globin vectors have demanding requirements including high-efficiency transduction at the HSC level and high-level, erythroid-specific expression with long-term pers...
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| Publicado en: | Nat Commun |
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| Autores principales: | , , , , , , , , , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
Nature Publishing Group UK
2019
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| Materias: | |
| Acceso en línea: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6775231/ https://ncbi.nlm.nih.gov/pubmed/31578323 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-019-12456-3 |
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