The DOI matches its Crossref record; 159 of the 159 references it cites were found in a registry or an index.
Strategies, design, and chemistry in siRNA delivery systems
Open accessAbstract
Emerging therapeutics that utilize RNA interference (RNAi) have the potential to treat broad classes of diseases due to their ability to reversibly silence target genes. In August 2018, the FDA approved the first siRNA therapeutic, called ONPATTRO™ (Patisiran), for the treatment of transthyretin-mediated amyloidosis. This was an important milestone for the field of siRNA delivery that opens the door for additional siRNA drugs. Currently, more than 20 small interfering RNA (siRNA)-based therapies are in clinical trials for a wide variety of diseases including cancers, genetic disorders, and viral infections. To maximize therapeutic benefits of siRNA-based drugs, a number of chemical strategies have been applied to address issues associated with efficacy, specificity, and safety. This review focuses on the chemical perspectives behind non-viral siRNA delivery systems, including siRNA synthesis, siRNA conjugates, and nanoparticle delivery using nucleotides, lipids, and polymers. Tracing and understanding the chemical development of strategies to make siRNAs into drugs is important to guide development of additional clinical candidates and enable prolonged success of siRNA therapeutics.
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Cited by
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Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics
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Therapeutic siRNA: State-of-the-Art and Future Perspectives
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Lipid nanoparticle technology for therapeutic gene regulation in the liver
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Non-viral vectors for RNA delivery
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Metadata
- Authors
- Dong, Y., Siegwart, D. J., & Anderson, D. G.
- Venue
- Advanced Drug Delivery Reviews
- Publisher
- Elsevier BV
- Published
- 2019-04
- Volume
- 144
- Pages
- 133-147
- ISSN
- 0169-409X (ISSN-L)
- OpenAlex
- W2945507495
- Cited by
- 572
- References
- 159
- FWCI
- 4.06
- Type
- journal article
- Language
- en
- Open access
- bronze
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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