治疗肺部囊性纤维化的黏液穿透型纳米基因载体的研究进展  

Progress of mucus penetrating nano gene carriers for the treatment of pulmonary cystic fibrosis

作　　者：方嘉宜 罗沛力 曾竟轩 裴庆澳 王文浩 黄郑炜 黄莹 潘昕[2] 吴传斌 FANG Jiayi;LUO Peili;ZENG Jingxuan;PEI Qingao;WANG Wenhao;HUANG Zhengwei;HUANG Ying;PAN Xin;WU Chuanbin(School of Pharmaceutical Sciences,Jinan University,Guangzhou 510032,China;School of Pharmaceutical Sciences,Sun Yat-sen University,Guangzhou 510006,China)

机构地区：[1]暨南大学药学院,广州510032 [2]中山大学药学院,广州510006

出　　处：《广东药科大学学报》2021年第4期132-137,共6页Journal of Guangdong Pharmaceutical University

基　　金：暨南大学大学生创新创业训练计划资助项目(CX20097)。

摘　　要：目前针对肺部囊性纤维化的疗法主要包括体位引流、抗生素联合祛痰药物和基因疗法等,其中基因疗法因能从根源上治疗疾病而独具优势。但是,在罹患肺部囊性纤维化后,患者肺部黏液发生病理变化,如黏液黏弹性增加、pH值降低等,使得普通基因载体难以穿透黏液到达病灶,导致基因疗法的治疗效果并不理想。为了克服这类患者的肺部黏液屏障,药剂学家提出了通过黏液穿透型基因载体进行药物递送的策略。有关研究热点包括调控粒径、化学修饰载体表面和共载辅助因子等。本文对肺黏液的组成特征、肺部囊性纤维化患者肺黏液的变化和黏液穿透型基因载体的设计策略及其穿透效果表征方式进行了综述,为用于治疗肺部囊性纤维化的黏液穿透型纳米基因载体的后续研究提供参考,促进此类纳米基因载体在基因疗法上的临床应用。At present,the treatment of pulmonary cystic fibrosis mainly includes postural drainage,antibiotics combined with expectorant drugs and gene therapy,among which gene therapy has unique advantages because it can treat the disease on the origin level.However,after suffering from pulmonary cystic fibrosis,pathological changes occur in the lung mucus of patients,such as the increase of mucus viscoelasticity and the decrease of pH value,which makes it difficult for ordinary gene carriers to penetrate the mucus to reach the lesions,resulting in the unsatisfactory therapeutic effect of gene therapy.In order to overcome the pulmonary mucus barrier in these patients,pharmacists have proposed a strategy of drug delivery through mucus penetrating gene carriers.The research focus includes controlling of particle size,chemical modification of carrier surface and co-loading of cofactors.This paper reviews the composition and characteristics of pulmonary mucus,the changes of pulmonary mucus in patients with pulmonary cystic fibrosis,the design strategies of mucus penetrating gene carriers and the characterization methods of their penetrating effect,so as to provide reference for the follow-up research of mucus penetrating nano gene carriers for the treatment of pulmonary cystic fibrosis and promote the clinical application of such nano gene carriers in gene therapy.

关 键 词：囊性纤维化 肺黏液 黏液穿透 纳米基因载体 

分 类 号：R943[医药卫生—药剂学]