Sickle Cell Disease: The Role of the Non-Viral Sleeping Beauty Transposon System Driven by the IHK Promoter in Inducing Sustained ?-globin expression and a Model Case Study

Abstract

Sickle cell disease is a relatively common disorder that affects approximately 100,000 people in the United States. More than 312,000 infants are born with this disease each year around the world, with 230,000 such births occurring annually in Sub-Saharan Africa alone. Sickle cell disease is dangerous not only because of the symptoms it presents, but because the majority of cases occur in developing parts of the world where treatment options are relatively nonexistent. The first chapter of this thesis outlines the disease in general, including the pathogenesis, diagnosis, symptoms, genetics, epidemiology, treatment, and clinical features of sickle cell disease. The second chapter is composed of a literature review that discusses the use of the Sleeping Beauty transposon system under the control of various molecular mechanisms to promote normal ?-globin protein expression. The specifics of these mechanisms, most notably the use of the IHK promoter, are further elucidated through experimental data.