Beta thalassemia gene therapy using lentiviral vectors

Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising to cure thalassemia. The goal of beta thalassemia gene therapy is to restore normal RBC production capacity in patients by suitable vector and correct inherited anemia. Virus therapy has recently been recognized as a promising new therapeutic approach in medical research. In this regard, the use of viruses as non-replicating gene therapy vectors or as replication viruses (oncolytic viruses) has provided attractive opportunities for viral therapy applications. Currently viral vectors have been used in nearly 70% of the clinical trials. In recent years, many researchers have designed erythroid-specific lentiviral vectors carrying the beta-globin gene that have obtained significant results. Their findings are presented in this study.