Department of Rehabilitation and Physiotherapy, Tangdu Hospital, The Fourth Military Medical University, Xi'an 710038, China.
Biochem Biophys Res Commun. 2012 Jul 27;424(2):203-7. doi: 10.1016/j.bbrc.2012.06.090. Epub 2012 Jun 26.
Transfecting foreign genes into target cells require certain vectors and specific techniques. With more new genes and gene targets for gene therapy are continually being discovered, which provide useful clues for the study of gene function and gene therapy for human disease. However, there still remain a number of important unresolved problems associated with the use of viral and non viral vectors or techniques, such as secondary toxicity, immune response, or low gene transfer efficiency. Therefore, efficient and safe approaches of gene delivery in vivo still need to be found for medical applications. Electroporation or ultrasound (US), which involving electrical pulses or a US field to increase cell membrane permeability, has shown to be an efficient, safe and simple non viral physical method of DNA delivery in vivo and alternative technique in the field of gene therapy. However, the high field strength or energy often required for electroporation or US can result in tissue damage, thus limit their widely clinical applications. In recent years, site-specific gene delivery by electroporation or US has aroused much attention, because of optimized protocols and novel devices using a lower field strength than conventional methods, which has shown high transfection efficiency with minimal tissue damage. In this paper, we reviewed the advancement in the field of electroporation and US for gene delivery, particularly by site-specific delivery into skeletal muscle for gene therapy and their applications in Alzheimer's disease (AD)'s treatment.
将外源基因转染到靶细胞中需要特定的载体和特定的技术。随着越来越多的新基因和基因靶点被发现用于基因治疗，这为基因功能的研究和人类疾病的基因治疗提供了有用的线索。然而，与使用病毒和非病毒载体或技术相关的一些重要未解决的问题仍然存在，例如二次毒性、免疫反应或低基因转移效率。因此，仍需要寻找有效的、安全的体内基因传递方法，以用于医疗应用。电穿孔或超声（US），涉及电脉冲或 US 场以增加细胞膜通透性，已被证明是一种有效的、安全的和简单的非病毒物理方法，可在体内进行 DNA 传递，是基因治疗领域的替代技术。然而，电穿孔或 US 通常需要高强度或能量，这可能导致组织损伤，从而限制了它们的广泛临床应用。近年来，电穿孔或 US 的靶向基因传递引起了广泛关注，因为优化的方案和使用低于传统方法的新型设备，显示出了高转染效率和最小的组织损伤。本文综述了电穿孔和 US 在基因传递领域的进展，特别是通过靶向骨骼肌进行基因治疗及其在阿尔茨海默病（AD）治疗中的应用。
