Beta Thalassemia Gene Therapy by Lentiviral Transfer of Beta-Globin Gene

Background and Aim: Beta thalassemia is a common monogenicdisorder caused by a partial or complete reduction of beta globin chainssynthesis. It is estimated that 1.5% of the global population (80 to 90million people) are carriers worldwide. Common treatments have theircomplication. So, a molecular approach, such as gene therapy for directhealthy beta-globin gene transmission, seems quite promising to curethalassemia. The goal of this study to design lentiviral vector carrying thehealthy beta-globin gene and study beta glob gene expression in goalcell lineMethods: For our purpose, we designed the DEST Lentiviral vector carriedthe normal beta-globin gene and its promoter and packaged lentivirus inLentiX-293 T cell line. Then targeted cells (K562 cells) was transducedby packaged lentivirus containing β-globin cassette. After transduction,β-globin protein expression level was determined by flow cytometryResults: Our results showed that we have successfully packaged andgenerated lentivirus in LentiX -293 T cell line and flow cytometryanalysis showed that beta globin protein expression in a treated cell wasincreased. Beta-globin gene expression in control and a treated cell wasachieved 23% and 85%, respectivelyConclusion: These data indicated that the vector used in this study canbe useful in gene therapy in the patient’s hematopoietic stem cell. Thefinal goal of this study is to examine designed vector in hematopoieticstem cells promising therapeutic strategy for genetic diseases like betathalassemia.

کلیدواژه ها:

Beta-thalassemia ، Gene therapy ، Lentiviral vector