[HTML][HTML] Expression of an anti-sickling β-globin in human erythroblasts derived from retrovirally transduced primitive normal and sickle cell disease hematopoietic cells
IH Oh, ME Fabry, RK Humphries, R Pawliuk… - Experimental …, 2004 - Elsevier
OBJECTIVE: Recent improvements in human β-globin vector design have fueled interest in
gene therapy approaches to the treatment of human thalassemia and sickle cell disease
(SCD). The present study was undertaken to determine whether human β-globin mRNA and
protein could be obtained in the erythroid progeny of more primitive human target cells
transduced with a retrovirus containing murine stem cell virus long terminal repeats, a
phosphoglycerate kinase promoter driving the expression of a green fluorescence protein …
gene therapy approaches to the treatment of human thalassemia and sickle cell disease
(SCD). The present study was undertaken to determine whether human β-globin mRNA and
protein could be obtained in the erythroid progeny of more primitive human target cells
transduced with a retrovirus containing murine stem cell virus long terminal repeats, a
phosphoglycerate kinase promoter driving the expression of a green fluorescence protein …
