TY - JOUR
T1 - Recent advances in nanotechnology approaches for non-viral gene therapy
AU - Jiang, Yihang
AU - Fan, Miaozhuang
AU - Yang, Zhenxu
AU - Liu, Xiaochen
AU - Xu, Zhourui
AU - Liu, Shikang
AU - Feng, Gang
AU - Tang, Shuo
AU - Li, Zhengzheng
AU - Zhang, Yibin
AU - Chen, Shilin
AU - Yang, Chengbin
AU - Law, Wing Cheung
AU - Dong, Biqin
AU - Xu, Gaixia
AU - Yong, Ken Tye
N1 - Funding Information:
This work was supported by the Natural National Science Foundation of China (31871442), Guangdong Natural Science Foundation (2021A1515012159, 2019A1515012163), The Startup Grant from The University of Sydney and The Postgraduate Innovation Development Fund Project of Shenzhen University (315-0000470832). We also thank the Instrument Analysis Centre of Shenzhen University for the assistance with imaging analysis. The Startup Grant from The University of Sydney (H3165).
Publisher Copyright:
© 2022 The Royal Society of Chemistry.
PY - 2022/12/21
Y1 - 2022/12/21
N2 - Gene therapy has shown great potential in the treatment of many diseases by downregulating the expression of certain genes. The development of gene vectors as a vehicle for gene therapy has greatly facilitated the widespread clinical application of nucleic acid materials (DNA, mRNA, siRNA, and miRNA). Currently, both viral and non-viral vectors are used as delivery systems of nucleic acid materials for gene therapy. However, viral vector-based gene therapy has several limitations, including immunogenicity and carcinogenesis caused by the exogenous viral vectors. To address these issues, non-viral nanocarrier-based gene therapy has been explored for superior performance with enhanced gene stability, high treatment efficiency, improved tumor-targeting, and better biocompatibility. In this review, we discuss various non-viral vector-mediated gene therapy approaches using multifunctional biodegradable or non-biodegradable nanocarriers, including polymer-based nanoparticles, lipid-based nanoparticles, carbon nanotubes, gold nanoparticles (AuNPs), quantum dots (QDs), silica nanoparticles, metal-based nanoparticles and two-dimensional nanocarriers. Various strategies to construct non-viral nanocarriers based on their delivery efficiency of targeted genes will be introduced. Subsequently, we discuss the cellular uptake pathways of non-viral nanocarriers. In addition, multifunctional gene therapy based on non-viral nanocarriers is summarized, in which the gene therapy can be combined with other treatments, such as photothermal therapy (PTT), photodynamic therapy (PDT), immunotherapy and chemotherapy. We also provide a comprehensive discussion of the biological toxicity and safety of non-viral vector-based gene therapy. Finally, the present limitations and challenges of non-viral nanocarriers for gene therapy in future clinical research are discussed, to promote wider clinical applications of non-viral vector-based gene therapy.
AB - Gene therapy has shown great potential in the treatment of many diseases by downregulating the expression of certain genes. The development of gene vectors as a vehicle for gene therapy has greatly facilitated the widespread clinical application of nucleic acid materials (DNA, mRNA, siRNA, and miRNA). Currently, both viral and non-viral vectors are used as delivery systems of nucleic acid materials for gene therapy. However, viral vector-based gene therapy has several limitations, including immunogenicity and carcinogenesis caused by the exogenous viral vectors. To address these issues, non-viral nanocarrier-based gene therapy has been explored for superior performance with enhanced gene stability, high treatment efficiency, improved tumor-targeting, and better biocompatibility. In this review, we discuss various non-viral vector-mediated gene therapy approaches using multifunctional biodegradable or non-biodegradable nanocarriers, including polymer-based nanoparticles, lipid-based nanoparticles, carbon nanotubes, gold nanoparticles (AuNPs), quantum dots (QDs), silica nanoparticles, metal-based nanoparticles and two-dimensional nanocarriers. Various strategies to construct non-viral nanocarriers based on their delivery efficiency of targeted genes will be introduced. Subsequently, we discuss the cellular uptake pathways of non-viral nanocarriers. In addition, multifunctional gene therapy based on non-viral nanocarriers is summarized, in which the gene therapy can be combined with other treatments, such as photothermal therapy (PTT), photodynamic therapy (PDT), immunotherapy and chemotherapy. We also provide a comprehensive discussion of the biological toxicity and safety of non-viral vector-based gene therapy. Finally, the present limitations and challenges of non-viral nanocarriers for gene therapy in future clinical research are discussed, to promote wider clinical applications of non-viral vector-based gene therapy.
UR - http://www.scopus.com/inward/record.url?scp=85141235847&partnerID=8YFLogxK
U2 - 10.1039/d2bm01001a
DO - 10.1039/d2bm01001a
M3 - Review article
C2 - 36222758
AN - SCOPUS:85141235847
SN - 2047-4830
VL - 10
SP - 6862
EP - 6892
JO - Biomaterials Science
JF - Biomaterials Science
IS - 24
ER -