Evaluation of the Efficacy of Lentiviral Vectors in Gene Therapy of Beta-thalassemia Patients: A Systematic Review

Authors

1 Student Research Committee, Golestan University of Medical Sciences, Gorgan, Iran.

2 Gorgan Congenital Malformations Research Center, Golestan University of Medical Sciences, Gorgan, Iran AND Stem Cell Research Center, Golestan University of Medical Sciences, Gorgan, Iran AND Ischemic Disorders Research Center, Golestan University of Medical Sciences, Gorgan, Iran.

Abstract

Background
Beta thalassemiais a genetic blood abnormality identified through mutations, which reduce the synthesis of the ß-globin chain. Gene therapy through Lentiviral vectors have cured many of genetic disorders. The purpose of this study was to investigate the efficacy of lentiviral vectors in treatment of ß-thalassemia as a novel approach for sustained treatment and prevention of recurrent blood transfusion that has many adverse effects on the patients.
Materials and Methods
In this systematic review, a systematic search of online databases (Web of Science, Scopus, and Medline) with no language restriction between 2012 and 2020 using the combination related keywords of Mesh included (Thalassemia OR Beta thalassemia OR β_thalassemia OR BTM) AND (Lentivirus) AND (Genetic therapy OR Gene therapy). All valuable data was allocated, and two independent researchers considered all articles.
Results
Finally, 20 articles that met the criteria were selected. Articles were quantitative (n=16), and qualitative (n=4), their year of publication varied from 2012 to 2020. Results showed that the main outcome of the desired survey was therapeutic treatments for thalassemia worldwide. While highly compatible donors for allogeneic bone marrow transplantation are accessible to less than 30% of all patients, gene therapy has emerged as a holistic and practical approach for the remaining 70%.
Conclusion
Nowadays, with the development of gene therapy, there is hope for a cure for genetic diseases. The successes of gene therapy techniques can bring laboratory methods of gene therapy a step closer to clinical and general use as a common treatment for genetic defects.

Keywords