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A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction

We show that lentiviral delivery of human γ-globin gene under β-globin regulatory control elements in hematopoietic stem cells (HSCs) results in sufficient postnatal fetal hemoglobin (HbF) expression to correct sickle cell anemia (SCA) in the Berkeley “humanized” sickle mouse. Upon de-escalating the...

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Autors principals: Perumbeti, Ajay, Higashimoto, Tomoyasu, Urbinati, Fabrizia, Franco, Robert, Meiselman, Herbert J., Witte, David, Malik, Punam
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2009
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2723013/
https://ncbi.nlm.nih.gov/pubmed/19474450
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2009-01-201863
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