Ren Xianyue, Yang Liqun, Liang Xuan, Liu Zhenzhen, Deng Yubin
Laboratory of Research Center for Translational Medicine, the First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, Guangdong, China.
Sheng Wu Gong Cheng Xue Bao. 2013 May;29(5):568-77.
Gene therapy has been considered as a promising method for treatment of many diseases, such as acquired and genetic diseases. At present, there are two major vehicles for gene delivery including viral vectors and nonviral vectors. Viral vectors appear as high gene transfection efficiency, but some deficiencies such as inflammatory responses, recombination and mutagenesis have limited their use. On account of low pathogenicity, safety and cost-effectiveness, nonviral vectors have been attracted much attention. Cationic polymers are one of the nonviral vectors which have been widely studied. This review focuses on the structure of the cationic polymers and the interaction mechanism between the vector and DNA. We try to provide a framework for the future design and synthesis of nonviral vectors with high transfection efficiency and low toxicity for gene therapy.
基因治疗已被视为治疗许多疾病的一种有前景的方法，如后天性疾病和遗传性疾病。目前，基因递送有两种主要载体，包括病毒载体和非病毒载体。病毒载体表现出高基因转染效率，但一些缺陷，如炎症反应、重组和诱变，限制了它们的使用。由于低致病性、安全性和成本效益，非病毒载体受到了广泛关注。阳离子聚合物是已被广泛研究的非病毒载体之一。本文综述聚焦于阳离子聚合物的结构以及载体与DNA之间的相互作用机制。我们试图为未来设计和合成用于基因治疗的高转染效率和低毒性的非病毒载体提供一个框架。
