LENTIVIRAL GENE ADDITION AND GENOME EDITING IN THALASSEMIA: A NEW ERA OF PRECISION MEDICINE

Abstract

Thalassemia is an inherited blood disorder occur due to a mutation that affect hemoglobin production in our body. It is classified as alpha or beta depending upon whether this mutation affect alpha chain or beta chain. It can be treated by inserting a functional gene or by repairing the faulty gene within the hematopoietic stem cell. Early gene transfer efforts were not successful because of limited delivery vector and insufficiently known knowledge. Recent research has developed strategies that address these challenges, including the use of lentiviral vector. Now researchers are focused on precise correction of mutant gene, modifying DNA sequences, globin gene mutation and manipulating the translocation and RNA splicing to acquire the balanced globin synthesis. In this review the place of interest is to highlight the present gene therapy approaches, including lentiviral vector delivery and comparison with emerging gene editing approaches.