生物大分子核酸药物制剂的研究现状与展望  

Research Status and Prospect of Therapeutic Nucleic Acid Formulations

作　　者：杜子秀[1] 

机构地区：[1]上海交通大学药学院,上海200240

出　　处：《药学进展》2015年第3期188-192,共5页Progress in Pharmaceutical Sciences

基　　金：国家自然科学基金(No.30901881);上海市闵行区2013年度产学研合作计划项目(No.20121012634480)

摘　　要：目前生物大分子核酸药物研发亟待突破的瓶颈是,如何使核酸药物能克服生物学屏障,实现体内有效输送。无生物安全隐患并具低免疫原性、高基因包封能力和易于制备的非病毒载体仍存在输送效率低和化学毒性大等缺陷,其临床应用受到限制。介绍核酸药物的研发现状,主要对非病毒核酸载体的研究现状及发展动态进行总结性回顾分析,并指出,虽然非病毒载体尚存在不足之处,但其自身优势仍使其具有成为未来核酸药物输送体系主体的广阔应用前景。Currently, the bottleneck that desperately needs to break through in nucleic acid drug development is how to overcome biological barriers in nucleic acid delivery in vivo. Despite the advantages of less biological toxicity, low immunogenicity, high capability of gene encapsulation and easy preparation, non-viral vectors are still restricted clinically by high chemical toxicity and low delivery efficiency. In this article, the present situation of nucleic acid drug development was introduced, with emphasis on retrospective analysis of the research status and development trend of non-viral vectors for nucleic acid drugs. It was pointed out that non-viral vectors were still promising delivery systems for nucleic acid drugs in the future despite their various limitations.

关 键 词：核酸药物 非病毒载体 聚合物 脂质体 体内输送 生物学屏障 

分 类 号：R94[医药卫生—药剂学]