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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Udgivet i:Nat Commun
Main Authors: 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.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2019
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Article
Online adgang: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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