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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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Detalles Bibliográficos
Publicado en:Nat Commun
Autores principales: Uchida, Naoya, Hsieh, Matthew M., Raines, Lydia, Haro-Mora, Juan J., Demirci, Selami, Bonifacino, Aylin C., Krouse, Allen E., Metzger, Mark E., Donahue, Robert E., Tisdale, John F.
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group UK 2019
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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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