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Research Status and Prospect of Non-Viral Vectors Based on siRNA: A Review

Gene therapy has attracted much attention because of its unique mechanism of action, non-toxicity, and good tolerance, which can kill cancer cells without damaging healthy tissues. siRNA-based gene therapy can downregulate, enhance, or correct gene expression by introducing some nucleic acid into pa...

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Autores principales: Tong, Liangnan, Liu, Danqing, Cao, Zhiyue, Zheng, Nannan, Mao, Chenchen, Liu, Shujuan, He, Liangcan, Liu, Shaoqin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9962405/
https://www.ncbi.nlm.nih.gov/pubmed/36834783
http://dx.doi.org/10.3390/ijms24043375
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author Tong, Liangnan
Liu, Danqing
Cao, Zhiyue
Zheng, Nannan
Mao, Chenchen
Liu, Shujuan
He, Liangcan
Liu, Shaoqin
author_facet Tong, Liangnan
Liu, Danqing
Cao, Zhiyue
Zheng, Nannan
Mao, Chenchen
Liu, Shujuan
He, Liangcan
Liu, Shaoqin
author_sort Tong, Liangnan
collection PubMed
description Gene therapy has attracted much attention because of its unique mechanism of action, non-toxicity, and good tolerance, which can kill cancer cells without damaging healthy tissues. siRNA-based gene therapy can downregulate, enhance, or correct gene expression by introducing some nucleic acid into patient tissues. Routine treatment of hemophilia requires frequent intravenous injections of missing clotting protein. The high cost of combined therapy causes most patients to lack the best treatment resources. siRNA therapy has the potential of lasting treatment and even curing diseases. Compared with traditional surgery and chemotherapy, siRNA has fewer side effects and less damage to normal cells. The available therapies for degenerative diseases can only alleviate the symptoms of patients, while siRNA therapy drugs can upregulate gene expression, modify epigenetic changes, and stop the disease. In addition, siRNA also plays an important role in cardiovascular diseases, gastrointestinal diseases, and hepatitis B. However, free siRNA is easily degraded by nuclease and has a short half-life in the blood. Research has found that siRNA can be delivered to specific cells through appropriate vector selection and design to improve the therapeutic effect. The application of viral vectors is limited because of their high immunogenicity and low capacity, while non-viral vectors are widely used because of their low immunogenicity, low production cost, and high safety. This paper reviews the common non-viral vectors in recent years and introduces their advantages and disadvantages, as well as the latest application examples.
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spelling pubmed-99624052023-02-26 Research Status and Prospect of Non-Viral Vectors Based on siRNA: A Review Tong, Liangnan Liu, Danqing Cao, Zhiyue Zheng, Nannan Mao, Chenchen Liu, Shujuan He, Liangcan Liu, Shaoqin Int J Mol Sci Review Gene therapy has attracted much attention because of its unique mechanism of action, non-toxicity, and good tolerance, which can kill cancer cells without damaging healthy tissues. siRNA-based gene therapy can downregulate, enhance, or correct gene expression by introducing some nucleic acid into patient tissues. Routine treatment of hemophilia requires frequent intravenous injections of missing clotting protein. The high cost of combined therapy causes most patients to lack the best treatment resources. siRNA therapy has the potential of lasting treatment and even curing diseases. Compared with traditional surgery and chemotherapy, siRNA has fewer side effects and less damage to normal cells. The available therapies for degenerative diseases can only alleviate the symptoms of patients, while siRNA therapy drugs can upregulate gene expression, modify epigenetic changes, and stop the disease. In addition, siRNA also plays an important role in cardiovascular diseases, gastrointestinal diseases, and hepatitis B. However, free siRNA is easily degraded by nuclease and has a short half-life in the blood. Research has found that siRNA can be delivered to specific cells through appropriate vector selection and design to improve the therapeutic effect. The application of viral vectors is limited because of their high immunogenicity and low capacity, while non-viral vectors are widely used because of their low immunogenicity, low production cost, and high safety. This paper reviews the common non-viral vectors in recent years and introduces their advantages and disadvantages, as well as the latest application examples. MDPI 2023-02-08 /pmc/articles/PMC9962405/ /pubmed/36834783 http://dx.doi.org/10.3390/ijms24043375 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Tong, Liangnan
Liu, Danqing
Cao, Zhiyue
Zheng, Nannan
Mao, Chenchen
Liu, Shujuan
He, Liangcan
Liu, Shaoqin
Research Status and Prospect of Non-Viral Vectors Based on siRNA: A Review
title Research Status and Prospect of Non-Viral Vectors Based on siRNA: A Review
title_full Research Status and Prospect of Non-Viral Vectors Based on siRNA: A Review
title_fullStr Research Status and Prospect of Non-Viral Vectors Based on siRNA: A Review
title_full_unstemmed Research Status and Prospect of Non-Viral Vectors Based on siRNA: A Review
title_short Research Status and Prospect of Non-Viral Vectors Based on siRNA: A Review
title_sort research status and prospect of non-viral vectors based on sirna: a review
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9962405/
https://www.ncbi.nlm.nih.gov/pubmed/36834783
http://dx.doi.org/10.3390/ijms24043375
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