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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。
Targeted cationic polymers, as non-viral vectors, can be targeted to the brain by carrying gene drugs and can be actively targeted through ligand-receptor specific binding mechanisms, which can effectively improve the expression of foreign genes in the brain and exert a therapeutic effect. The author of its current into the brain transport problems and solutions to elaborate, for the brain-targeted gene delivery system to provide a reference. Methods According to the main problems of gene carriers, the research on cationic polymers as brain gene delivery vectors was analyzed and summarized from the aspects of vector construction, ligand modification, stability improvement, transfection efficiency and toxicity reduction. RESULTS AND CONCLUSION Cationic polymers have high drug-loading properties as brain-targeting gene carriers, but they also have some problems such as low transfection efficiency, large cytotoxicity, and unclear targeting in cells. These problems can be solved by structural modification of polymers, Targeted modification and other strategies to improve. Cationic polymers, as a relatively safe and efficient non-viral gene delivery vector, can provide an effective route of administration for gene therapy of CNS diseases.