A LENTIVIRAL VECTOR CONFERRING COREGULATED, ERYTHROID-SPECIFIC EXPRESSION OF γ-GLOBIN AND shRNA SEQUENCES TO BCL11A FOR THE TREATMENT OF SICKLE CELL DISEASE
Sickle cell disease (SCD) is a severe hemoglobin disorder caused by co-inheritance of a single mutation in the β-globin gene of adult hemoglobin (HbA; α2β2). This alteration leads to the formation of sickle hemoglobin (HbS; α2βS2) and deformed, sickle-shaped red blood cells (RBCs). Sickle RBCs obstr...
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OpenSIUC
2016
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Online Access: | https://opensiuc.lib.siu.edu/theses/1995 https://opensiuc.lib.siu.edu/cgi/viewcontent.cgi?article=3009&context=theses |