91桃色

Abstract

Thalassemia is a genetic disease caused by frameshift mutation of gene on chromosome number 11 (β-thalassemia) or 16 (α-thlalassemia) resulting in reduce or abolish formation of the β- globin and α-globin chain respectively that form hemoglobin. This promotes formation of abnormal hemoglobin molecules causing anemia. Treatments of thalassemia involve: blood transfusion, iron chelation therapy and bone-marrow transplantation. These methods have many complications like high risk of infection, negative response of immune system against the transplant and transfused blood, weak immune system due to immune suppressive drugs and these are temporary, time taking and expensive. In this context, we can use our understanding of gene therapy through Lentiviral vector (LV). LV carry genes that promote host cell division, it reproduce inside host cell indefinitely without killing it and carry strong promoters for viral gene expression which can be subverted to control the expression of transgenes. Transferring functional gene to hematopoietic stem cells in bone marrow through LV can permanently produce normal hemoglobin. LV has shown promise in curing the thalassemia in mouse model, although it causes insertional mutagenesis. If we design a regulator which control expression or activation of oncogenes then it would be a possible, promising and permanent cure of thalessemia.

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