Abstract

A REVIEW ON GENE THERAPY FOR HEAMOGLOBIN DISORDERS

Farhanban S. H.*, Meenu A. P., Soumya R. V. and Prasobh G. R.

ABSTRACT

Gene therapy is an excellent way for relieving hemoglobin disorders. They can be done by either insertion or deletion. Thus it is considered as a best option for sickle cell anaemia and thalassemia. Hemoglobinopathies are genetic disorders affecting the natural hemoglobin synthesis. Hemoglobin is a protein attached to the surface of erythrocytes, which plays a crucial role in the transport of gases(mainly O2 and Co2). Thalassemia and sickle cell anaemia are the major two disorders coming under hemoglobinopathies. Thalassemia is due to under production of alpha and beta chain of the hemoglobin whereas the sickle cell anemia is due to a point mutation in beta chain which leads to polymerization of hemoglobin causing the structural deformity to the RBC. The modern medicine evolves some Special techniques for the permanent cure of these hemoglobinopathies. It includes hemopoietic stem cell transplant. This nees HLA compatibility between donor and recipient. Whereas in gene therapy modified hemopoietic stem cells are used, which does not require HLA compatibility. The vector mediated gene transfer is highly efficient in the management of hemoglobinopathies. The commonly used vectors include marine Moloney leukemia virus, HIV 1 and foamy virus, which all are associated with locus control region. Whereas Gamma retrovirus is used as vector there is no need of the locus controlling region elements, they utilizes long terminal repeat for the transgene expression.Despite their success rates, there is also a chance of mutagenecity. In lentivirus based gene therapy we use the ability of HIV-1 to transduce non deciding /quescent cell. They also have some safety problems such as causing mutagenicity. So the HIV regulatory genes and accessory genes from plasmid are removed.Transposases are another important method used. Transposases are used for integration of desired genome into target cells.

Keywords: Gene; Hemoglobinopathies; Sickle cell anemia; Thalassemia; Hemoglobin; Therapy; Disorders.