Emerging non-viral vectors for gene delivery

被引:92
作者
Wang, Chenfei [1 ]
Pan, Chaolan [1 ]
Yong, Haiyang [2 ]
Wang, Feifei [3 ]
Bo, Tao [4 ]
Zhao, Yitong [5 ]
Ma, Bin [2 ]
He, Wei [5 ]
Li, Ming [1 ]
机构
[1] Fudan Univ, Natl Childrens Med Ctr, Dept Dermatol, Childrens Hosp, Shanghai 201102, Peoples R China
[2] Xi An Jiao Tong Univ, Sch Chem Engn & Technol, Xian 710049, Shaanxi, Peoples R China
[3] Air Force Med Univ, Dept Anesthesiol, Affiliated Hosp 2, Xian 710032, Shaanxi, Peoples R China
[4] Fudan Univ, Sch Basic Med Sci, Shanghai 200032, Peoples R China
[5] Anhui Univ Sci & Technol, Sch Med, Huainan 232000, Anhui, Peoples R China
关键词
Gene therapy; Gene delivery process; Non-viral gene vectors; Nanomaterials; Lipid nanoparticles; POLY(BETA-AMINO ESTERS); IN-VITRO; SURFACE-CHEMISTRY; NANOPARTICLE SIZE; POLYMER STRUCTURE; SERUM STABILITY; MESSENGER-RNA; EFFICIENT; DNA; TRANSFECTION;
D O I
10.1186/s12951-023-02044-5
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
Gene therapy holds great promise for treating a multitude of inherited and acquired diseases by delivering functional genes, comprising DNA or RNA, into targeted cells or tissues to elicit manipulation of gene expression. However, the clinical implementation of gene therapy remains substantially impeded by the lack of safe and efficient gene delivery vehicles. This review comprehensively outlines the novel fastest-growing and efficient non-viral gene delivery vectors, which include liposomes and lipid nanoparticles (LNPs), highly branched poly(& beta;-amino ester) (HPAE), single-chain cyclic polymer (SCKP), poly(amidoamine) (PAMAM) dendrimers, and polyethyleneimine (PEI). Particularly, we discuss the research progress, potential development directions, and remaining challenges. Additionally, we provide a comprehensive overview of the currently approved non-viral gene therapeutics, as well as ongoing clinical trials. With advances in biomedicine, molecular biology, materials science, non-viral gene vectors play an ever-expanding and noteworthy role in clinical gene therapy.
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页数:18
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