Institute of Tissue Regeneration Engineering, Dankook University, Cheonan 330-714, South Korea.
Nanomedicine (Lond). 2013 Nov;8(11):1875-91. doi: 10.2217/nnm.13.165.
Over the past decade, stem cells have been considered to be a promising resource to cure and regenerate damaged or diseased tissues with research extending from basic studies to clinical application. Furthermore, genetically modified stem cells have the potential to reduce tumorigenic risks and achieve safe tissue formation. Recent advances in genetic modification of stem cells have rendered these cells more accessible and stable. The successful genetic modification of stem cells relies heavily on designing vector systems, either viral or nonviral vectors, which can efficiently deliver therapeutic genes to the cells with minimum toxicity. Currently, viral vectors showing high transfection efficiencies still raise safety issues, whereas safer nonviral vectors exhibit extremely poor transfection in stem cells. Here, we attempt to review and discuss the main factors raising concern in previous reports, and devise strategies to solve the issues in gene delivery systems for successful stem cell-targeting regenerative therapy.
在过去的十年中,干细胞被认为是一种有前途的资源,可以治愈和再生受损或患病的组织,研究范围从基础研究扩展到临床应用。此外,基因修饰的干细胞具有降低致瘤风险和实现安全组织形成的潜力。干细胞基因修饰的最新进展使得这些细胞更容易获得和更稳定。干细胞的成功基因修饰在很大程度上依赖于设计载体系统,无论是病毒载体还是非病毒载体,这些载体系统可以有效地将治疗基因传递到细胞中,同时毒性最小。目前,表现出高转染效率的病毒载体仍然存在安全性问题,而更安全的非病毒载体在干细胞中的转染效率极低。在这里,我们试图回顾和讨论以前报道中引起关注的主要因素,并设计策略来解决基因传递系统中的问题,以实现针对干细胞的靶向再生治疗的成功。