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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Detaylı Bibliyografya
Yayımlandı:Nat Commun
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group UK 2019
Konular:
Article
Online Erişim: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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