Emerging non-viral vectors for gene delivery

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 imp...

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Veröffentlicht in:Journal of nanobiotechnology Jg. 21; H. 1; S. 1 - 18
Hauptverfasser: Wang, Chenfei, Pan, Chaolan, Yong, Haiyang, Wang, Feifei, Bo, Tao, Zhao, Yitong, Ma, Bin, He, Wei, Li, Ming
Format: Journal Article
Sprache:Englisch
Veröffentlicht: London BioMed Central 17.08.2023
BioMed Central Ltd
Springer Nature B.V
BMC
Schlagworte:
Adsorption
Advanced Non-viral Delivery Systems in Tissue Engineering
Analysis
Biotechnology
Chain branching
Chemistry
Chemistry and Materials Science
Clinical trials
Cytoplasm
Cytotoxicity
Dendrimers
Deoxyribonucleic acid
DNA
Efficiency
Expression vectors
Gene delivery process
Gene expression
Gene therapy
Gene transfer
Genes
Genetic engineering
Genetic research
Genetic vectors
Lipid nanoparticles
Lipids
Materials science
Messenger RNA
Molecular biology
Molecular Medicine
Nanomaterials
Nanoparticles
Nanotechnology
Non-viral gene vectors
Physiology
Polyethylene glycol
Polyethyleneimine
Polymers
Proteins
Review
RNA
Vaccines
Vectors (Biology)
China
Nanomaterials
Gene delivery process
Gene therapy
Non-viral gene vectors
Lipid nanoparticles
ISSN:1477-3155, 1477-3155
Online-Zugang:Volltext
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Zusammenfassung: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(β-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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ISSN:1477-3155
1477-3155
DOI:10.1186/s12951-023-02044-5