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Research Progress of nucleic acid delivery vectors for gene therapy

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Abstract

Gene therapy has broad prospects as an effective treatment for some cancers and hereditary diseases. However, DNA and siRNA are easily degraded in vivo because of their biological activities as macromolecules, and they need the effective transmembrane delivery carrier Selecting the appropriate carrier for delivery will allow nucleic acid molecules to reach their site of action and enhance delivery efficiency. Currently used nucleic acid delivery vectors can be divided into two major categories: viral and non-viral vectors. Viral carrier transport efficiency is high, but there are safety issues. Non-viral vectors have attracted attention because of their advantages such as low immunogenicity, easy production, and non-tumorigenicity. The construction of safe, effective, and controllable vectors is the focus of current gene therapy research. This review presents the current types of nucleic acid delivery vehicles, which focuses on comparing their respective advantages and limitations, and proposes a novel delivery system, RNTs, a novel nanomolecular material, introducing the characteristics and nucleic acid delivery process of RNTs and their latest applications.

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Acknowledgments

The authors thank Guo Xiong and Zhang Feng for reviewing the manuscript. We thank M. Arico from Liwen Bianji, Edanz Group China (www.liwenbianji.cn/ac), for editing the English text of a draft of this manuscript. This review was supported by grants from the National Nature Science Foundation of China (No. 81171761), Shaanxi Natural Science Foundation (No. 2017SF-088), and Shaanxi Provincial Department of Education Fund (No: 18JK0667). The funding sources had no role in this publication.

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Y.J. and Z.L.X. were responsible for study conception and design, acquisition of data, analysis and interpretation of data, and drafting the manuscript. L.J.Z. critically revised the manuscript for important intellectual content. H.J. and B.X. were responsible for the analysis and interpretation of data. S.F. was responsible for study conception and design, acquisition, analysis and interpretation of data, and critical revision of the manuscript for important intellectual content. All authors read and approved the final version of the manuscript and had full access to all of the data in the study.

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Correspondence to Sun Fu.

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Jiao, Y., Xia, Z.L., Ze, L.J. et al. Research Progress of nucleic acid delivery vectors for gene therapy. Biomed Microdevices 22, 16 (2020). https://doi.org/10.1007/s10544-020-0469-7

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