%0 Journal Article
%T Induction of Fetal Hemoglobin In Vivo Mediated by a Synthetic ¦Ã-Globin Zinc Finger Activator
%A Fl¨¢via C. Costa
%A Halyna Fedosyuk
%A Renee Neades
%A Johana Bravo de Los Rios
%A Carlos F. Barbas III
%A Kenneth R. Peterson
%J Anemia
%D 2012
%I Hindawi Publishing Corporation
%R 10.1155/2012/507894
%X Sickle cell disease (SCD) and β-thalassemia patients are phenotypically normal if they carry compensatory hereditary persistence of fetal hemoglobin (HPFH) mutations that result in increased levels of fetal hemoglobin (HbF, γ-globin chains) in adulthood. Thus, research has focused on manipulating the reactivation of γ-globin gene expression during adult definitive erythropoiesis as the most promising therapy to treat these hemoglobinopathies. Artificial transcription factors (ATFs) are synthetic proteins designed to bind at a specific DNA sequence and modulate gene expression. The artificial zinc finger gg1-VP64 was designed to target the −117 region of the Aγ-globin gene proximal promoter and activate expression of this gene. Previous studies demonstrated that HbF levels were increased in murine chemical inducer of dimerization (CID)-dependent bone marrow cells carrying a human β-globin locus yeast artificial chromosome (β-YAC) transgene and in CD34
%U http://www.hindawi.com/journals/ane/2012/507894/