遗传增强
免疫原性
病毒载体
基因传递
纳米技术
载体(分子生物学)
计算生物学
生物
免疫系统
基因
材料科学
重组DNA
免疫学
遗传学
作者
Jianxiang Zhang,Xiaohui Li,Liping Lou,Xiaodong Li,Yong Jia,Zhe Jin,Yuxuan Zhu
出处
期刊:Fundamental biomedical technologies
日期:2011-01-01
卷期号:: 599-699
被引量:4
标识
DOI:10.1007/978-94-007-1248-5_21
摘要
Gene therapy has been considered to be a powerful approach for the prevention and/or treatment of a variety of diseases from genetic disorders, infections, to cancer. The success of gene therapy in the clinic is largely limited currently, mainly due to the lack of safe and efficient delivery vectors. Despite the high transfection efficiency, viral vectors encounter the vital toxicity issues and production problems. Increasing endeavors have been therefore directed towards the development of non-viral systems with the advantages of low immunogenicity and toxicity, ease in manufacturing and mass production, low cost, excellent stability, reduced vector size limitations, high flexibility regarding the size of transgenes to be delivered, and diverse chemical designs for constructing vectors with multiple functions. In this chapter, we summarized most of the synthetic non-viral systems currently employed for gene therapy, including lipid and polymer-based vectors, nanomaterials such as magnetic nanoparticles, quantum dots, gold/silica nanostructures, carbon nanotubes, calcium phosphate nanoparticles, and layered double hydroxides/clays, as well as multifunctional nanosystems among them. Particular attention has been paid on synthetic polymers and the related nanomedicines. Selected clinical trials of gene therapy using non-viral vectors as well as the future development in this rapidly growing field were briefly discussed.
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