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  • 标题:Evaluation of the Efficacy of Lentiviral Vectors in Gene Therapy of Beta-thalassemia Patients: A Systematic Review
  • 本地全文:下载
  • 作者:Saeed Dorgaleleh ; Ahmad Barahouie ; Farzad Dastaviz
  • 期刊名称:International Journal of Pediatrics
  • 印刷版ISSN:2345-5047
  • 电子版ISSN:2345-5055
  • 出版年度:2020
  • 卷号:8
  • 期号:5
  • 页码:11243-11250
  • DOI:10.22038/ijp.2020.47309.3839
  • 出版社:Mashhad University of Medical Sciences
  • 摘要: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 allogen e ic 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.
  • 关键词:Beta-Thalassemia; Lentiviral; Gene Therapy; vectors
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