Keywords : Gene therapy, Sickle Cell Anemia, Stem cells, lentivirus, β-globin
Project type: GBE-492 Genetic Engineering Project
I. INTRODUCTION, BACKGROUND and AIM
Sickle cell anemia is a devastating genetic disease which causes premature death. Sickle cell disease (SCD) is caused due to single amino acid change (glutamine to valine at position 6) in β chain of hemoglobin molecule, which causes the hemoglobin molecules to aggregate. These aggregated hemoglobin molecules make the red blood cells (RBCs) stiff and deformed (sickle shaped). These deformed cells block the normal blood flow. Although this disease is well-characterized, but no long-term treatment 1 is still available for this disease.
The discovery of the sickle ...