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Mesenchymal stem cells as carrier of the therapeutic agent in the gene therapy of blood disorders

机译:间充质干细胞的载体基因疗法的治疗代理血障碍

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Nonhematopoietic stem cells as a delivery platform of therapeutic useful genes have attracted widespread attention in recent years, owing to gained a long lifespan, easy separation, high proliferation, and high transfection capacity. Mesenchymal stem/ stromal cells (MSCs) are the choice of the cells for gene and cell therapy due to high self-renewal capacity, high migration rate to the site of the tumor, and with immune suppressive and anti-inflammatory properties. Hence, it has a high potential of safety genetic modification of MSCs for antitumor gene expression and has paved the way for the clinical application of these cells to target the therapy of cancers and other diseases. The aim of gene therapy is targeted treatment of cancers and diseases through recovery, change, or enhancement cell performance to the sustained secretion of useful therapeutic proteins and induction expression of the functional gene in intended tissue. Recent developments in the vectors designing leading to the increase and durability of expression and improvement of the safety of the vectors that overcome a lot of problems, such as durability of expression and the host immune response. Nowadays, gene therapy approach is used by MSCs as a delivery vehicle in the preclinical and the clinical trials for the secretion of erythropoietin, recombinant antibodies, coagulation factors, cytokines, as well as angiogenic inhibitors in many blood disorders like anemia, hemophilia, and malignancies. In this study, we critically discuss the status of gene therapy by MSCs as a delivery vehicle for the treatment of blood disorders. Finally, the results of clinical trial studies are assessed, highlighting promising advantages of this emerging technology in the clinical setting.
机译:Nonhematopoietic干细胞作为交付平台治疗有用的基因吸引了近年来广泛的关注,由于获得长寿命,容易分离,高扩散和高转染能力。间充质干细胞(msc) /基质细胞选择细胞的基因和细胞治疗高自我更新能力、高迁移率的肿瘤和免疫抑制和消炎作用。因此,它有一个高安全的遗传潜力修改的msc抗肿瘤基因表达式,并为临床铺平了道路应用这些细胞治疗目标癌症和其他疾病。治疗癌症和有针对性的治疗疾病通过复苏、改变或提高电池性能的持续分泌有用的治疗性蛋白质和归纳功能基因的表达组织。设计导致增加和耐用性的表达和改善的安全克服很多问题的向量,耐用性的表达和宿主的免疫响应。msc作为运载工具的基础和分泌的临床试验促红细胞生成素,重组抗体,凝血因子,细胞因子,以及血管生成抑制剂在许多血液疾病如贫血、血友病和恶性肿瘤。这项研究中,我们批判性讨论的状态基因治疗通过msc的运载工具血液疾病的治疗。临床试验研究的结果评估,强调有前途的优势新兴技术在临床设置。

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