聚乙烯亚胺纳米基因递送系统的研究进展  

Research advances of polyethylenimine-based nanosystems for gene delivery

作　　者：胡立中 李靖 郁川 杜煜 宋敏杰 张邦乐 HU Lizhong;LI Jing;YU Chuan;DU Yu;SONG Minjie;ZHANG Bangle(Animal Diseases&Public Health Engineering Research Center of Henan Province,Luoyang Vocational&Technical College,Henan Luoyang 471023,China;School of Pharmacy,Air Force Medical University,Shaanxi Xi’an 710032,China)

机构地区：[1]洛阳职业技术学院河南省动物疫病与公共卫生工程研究中心,河南洛阳471023 [2]空军军医大学药学院,陕西西安710032

出　　处：《中国医院药学杂志》2024年第7期853-858,共6页Chinese Journal of Hospital Pharmacy

基　　金：国家自然科学基金项目(编号:82072052);河南省高等学校重点科研项目(编号:24B350006)。

摘　　要：基因治疗在恶性肿瘤、感染性疾病、自身免疫性疾病、罕见病等重大难治性疾病的治疗中表现出巨大潜力。基因递送载体是基因治疗能否成功实施的关键所在,聚乙烯亚胺(PEI)是一种被广泛研究的阳离子基因递送载体,在不同细胞系和转染条件下均展现出稳定高效的基因转染效果,其中PEI25k更被视作基因转染的“黄金标准”。为解决PEI在基因递送中存在的体内转染效率低、细胞毒性大、靶向性低和负载基因溶酶体降解等问题,该文对基于PEI设计构建新型纳米递送系统用于基因治疗的研究进展进行综述,主要包括高相对分子质量线性PEI、多糖、亲水性的聚合物和右旋糖酐修饰的PEI,交联的低相对分子质量PEI,基于PEI的无机纳米递送载体以及基于PEI的药物与基因共递送载体系统,以期为进一步构建高效低毒的基因递送系统提供理论指导。Gene therapy has demonstrated great therapeutic potentials for such major refractory diseases as malignant tumors,infectious,autoimmune and rare disease,etc.Gene delivery vectors are essential for a successful implementation of gene therapy.Polythylenimine(PEI)is a widely studied cationic gene delivery vector,showing stable and efficient gene transfection in numerous cell lines and transfection conditions.PEI25k is regarded as a"gold standard"for gene transfection.Although PEI has broad application prospects in the field of gene delivery,there are still urgent problems to be tackled,including low transfection efficiency in vivo,high cytotoxicity,low targeting capability and lysosomal degradation of loaded gene.This review summarized the latest advances of designing novel polyethylenimine-based nanosystems for gene therapy,including high molecular weight linear PEI,PEI modified with polysaccharides,hydrophilic polymers or dextran,crosslinked low molecular weight branched PEI,PEI-based inorganic nanoparticles and co-delivery vectors of drug/gene based upon PEI.It was intended to provide theoretical rationales for further constructing gene delivery carriers with high efficiency and low toxicity.

关 键 词：聚乙烯亚胺 基因治疗 阳离子聚合物 非病毒载体 基因递送系统 

分 类 号：R945[医药卫生—微生物与生化药学]