Abstract：
Hematopoietic stem cell (HSC) gene therapy has shown potential as a therapeutic approach for thalassemia in recent years. However, a comparison of the varying gene therapy methods of HSC gene therapy in thalassemia has never been reviewed. This study aims to evaluate the utilization of HSC gene therapy approaches in animal models of thalassemia. A systematic review was conducted in five databases: PubMed, EBSCOHost, Science Direct, SCOPUS, and Proquest using a combination of the terms hematopoietic stem cell or hematopoietic stem cell or HSC, thalassemia, genetic therapy or gene therapy and animal model. Only journals published in English between 2008 and 2023 were included. This literature included six studies analyzing the use of HSC gene therapy in thalassemic mice models. The three outcomes being assessed in this review were globin levels, hematological parameters, and red blood cell (RBC) phenotypes. Gene therapy approaches for thalassemia using HSC showed significant improvement in β-globin levels and RBC phenotypes. Phenotypic improvements were also observed. These outcomes indicate good efficacy in gene therapy for thalassemia in mice models. Furthermore, more studies assessing the efficacy of HSC gene therapy in the human model should be done in future studies.
摘要：
近年来，造血干细胞（HSC）基因治疗已显示出作为地中海贫血治疗方法的潜力。然而,尚未对地中海贫血中HSC基因治疗的各种基因治疗方法进行比较。本研究旨在评估HSC基因治疗方法在地中海贫血动物模型中的应用。在五个数据库中进行了系统评价：PubMed，EBSCOHost,科学直接，Scopus,和Proquest使用造血干细胞或造血干细胞或HSC的组合，地中海贫血,基因治疗或基因治疗和动物模型。仅包括2008年至2023年之间以英语出版的期刊。该文献包括六项分析在地中海贫血小鼠模型中使用HSC基因疗法的研究。本次审查评估的三个结果是全球水平，血液学参数,和红细胞（RBC）表型。使用HSC的地中海贫血基因治疗方法显示β-珠蛋白水平和RBC表型的显着改善。还观察到表型改善。这些结果表明在小鼠模型中对地中海贫血的基因治疗具有良好的功效。此外，在未来的研究中,应该进行更多的研究来评估HSC基因治疗在人类模型中的疗效.
