Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34(+) cells after lentiviral vector-mediated gene transfer

β-Thalassemia major results from severely reduced or absent expression of the β-chain of adult hemoglobin (α(2)β(2);HbA). Increased levels of fetal hemoglobin (α(2)γ(2);HbF), such as occurs with hereditary persistence of HbF, ameliorate the severity of β-thalassemia, raising the potential for geneti...

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Autors principals: Wilber, Andrew, Hargrove, Phillip W., Kim, Yoon-Sang, Riberdy, Janice M., Sankaran, Vijay G., Papanikolaou, Eleni, Georgomanoli, Maria, Anagnou, Nicholas P., Orkin, Stuart H., Nienhuis, Arthur W., Persons, Derek A.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2011
Matèries:
Gene Therapy
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3062294/
https://ncbi.nlm.nih.gov/pubmed/21156846
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2010-08-300723
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