摘要
基因治疗为各种先天性或后天性疾病(如肿瘤)提供了一种新的治疗方式,因此应用前景广阔。由于裸基因易受到多种因素(如,酶降解、肾滤过作用引起的快速清除、不易被细胞摄取、从内涵体到细胞质的释放不充分等)的影响,应用受到严重限制。要成功的达到基因治疗的目的,开发安全有效的基因传递系统十分必要。随着基因传递系统的日益发展,对所开发的载体的安全性要求也越来越高。本文介绍生物相容性高分子,如壳聚糖、聚氨基酯以及它们的衍生物作为肿瘤基因治疗非病毒高分子载体的一些研究动态。
Gene therapy holds great promise in providing new approaches for the treatment of a large number of inherited and acquired diseases, such as cancer. Yet its widespread application has been restricted by naked genes vulnerable to enzyme degradation, the rapid clearance by renal filtration, the poor cellular uptake and the inefficient escape out of endosome into cytosol. A successful gene therapy mainly relies on the development of an efficient gene delivery system. The safe and efficient delivery of therapeutic genes to host cells and subsequent release in the target compartment in a therapeutic way represents a fundamental obstacle to the clinical success of gene therapy. Here, we summarize the recent researches concerning biocompatible polymers such as chitosan, poly （ amino ester） s and their derivatives as non-viral vectors for cancer gene therapy.
出处
《中国药科大学学报》
CAS
CSCD
北大核心
2013年第5期476-481,共6页
Journal of China Pharmaceutical University
