Integrase minus lentiviral vector: A suitable vector for β-thalassemia gene targeting

"nβ-Thalassemia is a congenital disorder caused by mutation in β-globin gene. The current therapies to treat β-thalassemia have many complications and limitations necessitating development of new curative methods. Recently, a great attention has been paid to gene therapy of β-thalassemia using lentiviral vectors. Although insertion of transgene into these vectors direct regulated expression of β-globin at therapeutic levels, lentiviruses unfavorably are integrated into the host-cell chromosomes. Consequently there is a key challenge for clinical application of lentiviral vectors in treatment of β-thalassemia. In order to overcome this problem, we have presented a novel hypothesis based on homologous recombination technology including a single negative double positive selection strategy for β-globin gene targeting. Because of no effect on other regions of the host genome, gene targeting with integrase minus lentiviral vectors seems an appropriate and safe method to correct genetic defects like β-thalassemia.