Protein nanocapsules based vectors for efficient gene transfection  

作　　者：Xuan Hu Song Wang Junjie Xu Yilong Yang Zhang Zhang Xiaolin Wang Xiaopeng Zhang Wei Chen 

机构地区：[1]Science and Technology on Advanced Ceramic Fibers and Composites Laboratory,National University of Defense Technology,Changsha 410073,China [2]Laboratory of Vaccine and Antibody Engineering,Beijing Institute of Biotechnology,Beijing 100071,China

出　　处：《Nano Research》2022年第1期264-271,共8页纳米研究（英文版）

基　　金：supported by the National Science and Technology Major Project(Nos.2018ZX09J18101 and 2019ZX09J19103);the National Natural Science Foundation of China(No.81703048).

摘　　要：Gene therapy employs exogenous nucleic acids to treat genetic diseases and disorders.The insufficient cytosolic delivery of genetic materials remains a major hurdle for effective gene therapy.To address this challenge,we have designed and synthesized various cationic protein nanocapsules that can efficiently condense nucleic acids via self-assembly.Through systematically investigating the gene transfection efficiency of these nanocapsules as delivery vectors,we find that nanocapsules,which were synthesized with hydrolyzable polymers containing tertiary amine groups,afford the highest transfection efficiency(〜80%),resulting in stable protein expression for over four days.The mechanistic study reveals that tertiary amine groups facilitate the endosomal escape of the nucleic acid-nanocapsule complexes after their cell internalization via endocytosis.The subsequent hydrolysis of the polymers triggers the cytosolic release of the nucleic acids,thereby prompting gene expression.Our results not only provide a new class of gene delivery vectors but also detail the parameters for future vector design.

关 键 词：protein nanocapsule self-assembly gene transfection cytosolic delivery degradation 

分 类 号：TB383[一般工业技术—材料科学与工程]