摘要
目的阳离子聚合物作为非病毒载体携载基因药物靶向于脑部,通过配体-受体特异性结合等机制实现主动靶向,可有效提高外源基因在脑组织的表达,发挥治疗作用。笔者针对其目前入脑转运存在的问题及解决策略进行阐述,为脑靶向基因递释系统的研究提供参考。方法针对目前基因载体存在的主要问题,从载体构建、配体修饰、提高稳定性和转染效率、降低毒性等方面对阳离子聚合物作为脑部基因传递载体的研究进行分析并总结。结果与结论阳离子聚合物作为脑靶向基因载体具有高载药特点,但还存在转染效率低、细胞毒性大、细胞内靶向不明确等问题,可通过聚合物的结构改造、载体系统的靶向性修饰等策略进行改善。阳离子聚合物作为一类相对安全、高效的非病毒基因载体,可以为中枢神经系统疾病的基因药物治疗提供有效的给药途径。
OBJECTIVE Cationic polymers can deliver gene into brain and improve the impression of exogenous gene in brain tis- sues. This review elaborates the problems and strategies of cationic polymers tansfering gene into brain by cationic polymers, and pro- vides a reference for further study. METHODS The literatures about cationic polymers were searched, and the progress of cationic polymers as gene carrier from several aspects were analyzed and summarized such as vector construction, ligands modification, efficient improvement and toxicity reduction. RESULTS AND CONCLUSION Cationic polymer vector has the advantage of high drug-load- ing, but it has to overcome the limits of BBB, high cell toxicity, low transfection and low targeting. It can be improved by structure re- construction, ligand modification, and so on. Cationic polymer is relatively safe and high efficient, so it is capable of providing efficient approach of gene delivery.
出处
《中国药学杂志》
CAS
CSCD
北大核心
2013年第20期1701-1705,共5页
Chinese Pharmaceutical Journal
关键词
阳离子聚合物
脑靶向
基因
cationic polymer
brain targeting
gene
