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
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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6775231/
https://www.ncbi.nlm.nih.gov/pubmed/31578323
http://dx.doi.org/10.1038/s41467-019-12456-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6775231/
https://www.ncbi.nlm.nih.gov/pubmed/31578323
http://dx.doi.org/10.1038/s41467-019-12456-3

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