Sung Y K, Kim S W
1Department of Pharmaceutics and Pharmaceutical Chemistry, University of Utah, Salt Lake City, UT 84112 USA.
2Center for Controlled Chemical Delivery, University of Utah, Salt Lake City, UT 84112 USA.
Biomater Res. 2019 Mar 12;23:8. doi: 10.1186/s40824-019-0156-z. eCollection 2019.
Gene delivery systems are essentially necessary for the gene therapy of human genetic diseases. Gene therapy is the unique way that is able to use the adjustable gene to cure any disease. The gene therapy is one of promising therapies for a number of diseases such as inherited disorders, viral infection and cancers. The useful results of gene delivery systems depend open the adjustable targeting gene delivery systems. Some of successful gene delivery systems have recently reported for the practical application of gene therapy.
The recent developments of viral gene delivery systems and non-viral gene delivery systems for gene therapy have briefly reviewed. The viral gene delivery systems have discussed for the viral vectors based on DNA, RNA and oncolytic viral vectors. The non-viral gene delivery systems have also treated for the physicochemical approaches such as physical methods and chemical methods. Several kinds of successful gene delivery systems have briefly discussed on the bases of the gene delivery systems such as cationic polymers, poly(L-lysine), polysaccharides, and poly(ethylenimine)s.
The goal of the research for gene delivery system is to develop the clinically relevant vectors such as viral and non-viral vectors that use to combat elusive diseases such as AIDS, cancer, Alzheimer, etc. Next step research will focus on advancing DNA and RNA molecular technologies to become the standard treatment options in the clinical area of biomedical application.
基因递送系统对于人类遗传疾病的基因治疗至关重要。基因治疗是利用可调控基因治愈任何疾病的独特方法。基因治疗是治疗多种疾病(如遗传性疾病、病毒感染和癌症)的有前景的疗法之一。基因递送系统的有效结果取决于可调控的靶向基因递送系统。最近报道了一些成功的基因递送系统用于基因治疗的实际应用。
简要回顾了用于基因治疗的病毒基因递送系统和非病毒基因递送系统的最新进展。讨论了基于DNA、RNA的病毒载体和溶瘤病毒载体的病毒基因递送系统。也探讨了非病毒基因递送系统的物理化学方法,如物理方法和化学方法。基于阳离子聚合物、聚(L - 赖氨酸)、多糖和聚(乙烯亚胺)等基因递送系统简要讨论了几种成功的基因递送系统。
基因递送系统的研究目标是开发临床上相关的载体,如用于对抗艾滋病、癌症、阿尔茨海默病等难治性疾病的病毒和非病毒载体。下一步研究将集中于推进DNA和RNA分子技术,使其成为生物医学应用临床领域的标准治疗选择。