Lentiviral vector containing beta-globin gene for beta thalassemia gene therapy

Beta thalassemia is a common monogenic disorder caused by partial or complete reduction of beta globin chains synthesis. In recent years allogeneic bone marrow transplantation (BMT) has been considered to be the successful cure for patients with major, however this restricted due limited number HLA-matched donors. Therefore, molecular approaches including gene therapy direct normal transmission seem quite promising thalassemia. The goal study was evaluate expression in K562 cell line, as model hematopoietic cell, transduced lentiviral vector carrying gene. For our purpose, we designed DEST Lentiviral (3rd generation) and its promoter packaged LentiX-293T line. line lentivirus containing β-globin cassette (pLenti-HBB). Bulk cells suspension form were diluted seeded into 96-well micro plate manual pipetting. After single expansion, protein level clones determined flowcytometry Western blotting. Our results showed that have successfully generated Flowcytometry analysis achieved 20.3% bulk population MOI = 20 74.4% F2 clone. These data indicated used efficient vitro could applied patient's stem cell. final examine therapeutic strategy sickle disease.