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

Abstract

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.

Inorganic nanocarriers for siRNA delivery for cancer treatments

“…Nevertheless of competent gene-silencing effects, cancer treatment using TpRNAs is limited due to their short body circulation time, targeting inability, poor target accumulation ability, instability, low cellular uptake, and etc [17,18]. To overcome the above hurdles, the following approaches are used in delivering TpRNAs-viral vectors and non-viral vectors [19,20]. Viral vectors are classified into (i) genedelivery vehicles (e.g.…”

Non-Viral Carriers for Nucleic Acids Delivery: Fundamentals and Current Applications

“…Currently, viral vectors are the most commonly used in clinical trials of gene therapy drugs [1]. However, along with the high delivery efficacy due to the life cycle of viruses, these vectors are immunogenic, and their use can lead to undesirable toxic effects for patients [2,3]. Liposomes, polycations, peptides, and inorganic compounds are examples of non-viral carriers.…”

Immune‐check blocking combination multiple cytokines shown curative potential in mice tumor model

“…Some studies have shown that although the viral vector has high transfection efficiency, its application is greatly limited due to the potential security risks, such as the occurrence of leukemia induced by some retroviruses, and the limited capacity of the viral vector 35 . In contrast, nonviral vectors have the advantages of low cost, simple preparation, easy mass production, high safety, and unlimited length of exogenous genes 36 . Plasmid vector is an attractive in vivo transfection vector in animals that can be injected directly into specific tissues and can achieve effective higher levels of gene expression in vivo.…”