Thalassemia: Possible Cure Through Gene Therapy Using Lentiviral Vector | 4751
Journal of Biotechnology & Biomaterials
Like us on:
Our Group organises 3000+ Global Conferenceseries Events every year across USA, Europe & Asia with support from 1000 more scientific Societies and Publishes 700+ Open Access Journals which contains over 50000 eminent personalities, reputed scientists as editorial board members.
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.
Peer Reviewed Journals
Make the best use of Scientific Research and information from our 700 + peer reviewed, Open Access Journals