Tissue Engineering and Regenerative Medicine, Nanobiotechnology Research Center, Baqiyatallah University of Medical Sciences, Tehran, Iran.
Department of Tissue Engineering and Applied Cell Sciences, School of Advanced Technologies in Medicine, Shahid-Beheshti University of Medical Sciences, Tehran, Iran.
Adv Exp Med Biol. 2018;1119:103-118. doi: 10.1007/5584_2018_256.
Regenerative medicine (RM) is a promising new field of medicine that has mobilized several new tools to repair or replace lost or damaged cells or tissues by stimulating natural regenerative mechanisms nearby cell and tissue-based therapy approaches. However, mesenchymal stem cell (MSC) based therapy has been shown to be safe and effective to a certain degree in multiple clinical trial studies (CTSs) of several diseases, in most MSC CTSs the efficacy of treatment has been reported low. Therefore, researchers have focused on efficacy enhancing of MSC to improve migratory and homing, survival, stemness, differentiation and other therapeutic applicable properties by using different approaches. Gene therapy is one of the experimental technique tools that uses genes to change cells for therapeutic and investigation purposes. In this study has been focused on genetically modified MSCs for use in RM with an emphasis on CTSs. We highlight the basic concept of genetic modifications and also discuss recent clinical studies aspects. Recently reviewed studies show that MSC therapy with assistant gene therapy can be used in cancer therapy, heart diseases, Fanconi anemia and several other diseases.
再生医学（RM）是医学领域的一个充满希望的新领域，它调动了多种新工具，通过刺激附近细胞和组织的自然再生机制，来修复或替换丢失或受损的细胞或组织。然而，间充质干细胞（MSC）为基础的治疗方法在多种疾病的多项临床试验研究（CTS）中已被证明在一定程度上是安全有效的，但在大多数 MSC CTS 中，治疗的疗效报告较低。因此，研究人员通过使用不同的方法，专注于提高 MSC 的疗效，以改善其迁移和归巢、存活、干性、分化和其他治疗应用特性。基因治疗是一种实验技术工具，它利用基因来改变细胞，以达到治疗和研究的目的。在这项研究中，重点是对用于 RM 的基因修饰 MSC，强调 CTS。我们强调了基因修饰的基本概念，并讨论了最近的临床研究方面。最近的综述研究表明，辅助基因治疗的 MSC 疗法可用于癌症治疗、心脏病、范可尼贫血症和其他几种疾病。
