This article forms part of a special issue - Advanced Biosafety Materials for Prevention and Theranostics of Biosafety Issues – covering the rapid detection and elimination of pathogenic microorganisms and the control of infectious diseases. For the full issue content see: https://www.sciencedirect.com/journal/biosafety-and-health/vol/4/issue/2
Nucleic acid therapeutics, which involve transferring exogenous genes inside target cells, are a promising clinical treatment option that can regulate gene expression at the transcriptional or post-transcriptional level.
Ideally, this kind of treatment modality will not lead to an unwanted immune response. Compared with traditional treatment methods, nucleic acid therapeutics can achieve prolonged and stable curative effects. As an emerging treatment method, nucleic acid therapeutics have played an increasingly important role in clinical settings for the treatment of various conditions, including infectious diseases, cancer, immune-related diseases, and monogenetic diseases.
To date, a large number of clinical trials have been conducted, and more than 30 nucleic acid drugs have been approved, highlighting the strong potential of this approach in clinical practice. Diverse carriers are used to protect nucleic acids from being degraded and to help them reach their targets accurately. However, some carriers are known to cause negative effects on the release and expression of nucleic acid drugs as well as adverse effects such as allergic reactions and accumulation in the liver. Therefore, biosafety assessment of delivery systems before their application in clinical settings is critical.
In this review, the authors describe different delivery systems for nucleic acid drugs and discuss their biosafety in both preclinical and clinical studies, with particular focus on the carriers themselves, drug administration method, and overall treatment of the disease.
- The delivery systems of nucleic acid drugs are summarized and biosafety assessments in clinical trials are discussed.
- Carriers of nucleic acid drugs, including viral vectors, liposomes, polymers, and carrier-free delivery systems, play a significant role in nucleic acid therapeutics.
- Nucleic acid drugs, coupled with different delivery systems, are used to treat various diseases.
- Depending on the properties of a particular drug carrier, nucleic acid therapeutics are administered via different methods in clinical settings.
Keywords: Nucleic acid therapeutics, Delivery system, Biosafety, Viral vector, Lipid, Carrier-free delivery
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Article reference: Zhimin Li, Li Zhang, Kai Jiang, Yijing Zhang, Yonglin Liu, Guang Hu, Jie Song, Biosafety assessment of delivery systems for clinical nucleic acid therapeutics, Biosafety and Health, Volume 4, Issue 2, 2022, Pages 105-117, ISSN 2590-0536, https://doi.org/10.1016/j.bsheal.2022.03.003.
Biosafety and Health