The DOI matches its Crossref record; 85 of the 85 references it cites were found in a registry or an index.
Current progress of siRNA/shRNA therapeutics in clinical trials
Open accessAbstract
Through a mechanism known as RNA interference (RNAi), small interfering RNA (siRNA) molecules can target complementary mRNA strands for degradation, thus specifically inhibiting gene expression. The ability of siRNAs to inhibit gene expression offers a mechanism that can be exploited for novel therapeutics. Indeed, over the past decade, at least 21 siRNA therapeutics have been developed for more than a dozen diseases, including various cancers, viruses, and genetic disorders. Like other biological drugs, RNAi‐based therapeutics often require a delivery vehicle to transport them to the targeted cells. Thus, the clinical advancement of numerous siRNA drugs has relied on the development of siRNA carriers, including biodegradable nanoparticles, lipids, bacteria, and attenuated viruses. Most therapies permit systemic delivery of the siRNA drug, while others use ex vivo delivery by autologous cell therapy. Advancements in bioengineering and nanotechnology have led to improved control of delivery and release of some siRNA therapeutics. Likewise, progress in molecular biology has allowed for improved design of the siRNA molecules. Here, we provide an overview of siRNA therapeutics in clinical trials, including their clinical progress, the challenges they have encountered, and the future they hold in the treatment of human diseases.
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Cited by
Non-viral vectors for gene-based therapy
Yin, H., Kanasty, R. L., Eltoukhy, A. A., Vegas, A. J., Dorkin, J. R., & Anderson, D. G. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3763 · Cited by 3,123
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Hijacking the E3 Ubiquitin Ligase Cereblon to Efficiently Target BRD4
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Self-citation
Metadata
- Authors
- Burnett, J. C., Rossi, J. J., & Tiemann, K.
- Venue
- Biotechnology Journal
- Publisher
- Wiley
- Published
- 2011-07-11
- Volume
- 6
- Issue
- 9
- Pages
- 1130-1146
- ISSN
- 1860-6768 (ISSN-L)
- 1860-7314
- OpenAlex
- W2008161734
- Cited by
- 444
- References
- 85
- FWCI
- 20.22
- Type
- journal article
- Language
- en
- Open access
- green
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