RNAi and small interfering RNAs in human disease therapeutic applications
Open accessAbstract
Small interfering RNAs (siRNAs) have been shown to effectively downregulate gene expression in human cells, giving them potential to eradicate disease. Prospects for clinical applications are discussed in this review, along with an overview of recent history and our current understanding of siRNAs used for therapeutic application in human diseases, such as cancer and viral infections. Over recent years, progress has been made in lipids, ligands, nanoparticles, polymers and viral vectors as delivery agents and for gene-based expression of siRNA to enhance the efficacy and specificity of these methods while at the same time reducing toxicity. It has become apparent that given the recent advances in chemistry and delivery, RNAi will soon prove to be an important and widely used therapeutic modality.
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siRNA therapy can be applied for treating GARDs by targeted silencing of the disease-causing genes.
“Therefore, siRNA therapy can be applied for treating GARDs by targeted silencing of the disease-causing genes.[50, 52]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [50]
siRNA enables efficient downregulation of specific gene expression through complementary-based mRNA cleavage.
“siRNA, based on the RNA interference (RNAi) technology,[50] enables efficient downregulation of specific gene expression through complementary-based mRNA cleavage.[51]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [50]
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- Authors
- Lares, M. R., Rossi, J. J., & Ouellet, D. L.
- Affiliations
- City Of Hope National Medical Center (US)
- Corresponding author
- Rossi, John J.
- Venue
- Trends in biotechnology
- Publisher
- Elsevier BV
- Published
- 2010-11
- Volume
- 28
- Issue
- 11
- Pages
- 570-579
- ISSN
- 0167-7799 (ISSN-L)
- PMC
- PMC2955826
- OpenAlex
- W1980161576
- Cited by
- 277
- References
- 79
- Citations by year
- 2026: 2 · 2025: 9 · 2024: 8 · 2023: 7 · 2022: 3 · 2021: 15 · 2020: 11 · 2019: 17 · 2018: 13 · 2017: 14 · 2016: 23 · 2015: 23 · 2014: 19 · 2013: 28 · 2012: 46
- FWCI
- 3.14
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Trends in biotechnology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Heart, Lung, and Blood Institute (R01HL074704)
- National Institute of Allergy and Infectious Diseases (R37AI029329, R01AI042552)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Keywords
- RNA interference, siRNA, siRNA therapeutics, gene silencing, siRNA delivery, lipid nanoparticles, viral vectors, polymers, ligands, cancer therapy, antiviral therapy, clinical translation
- MeSH terms
- Biological Products, Humans, Genetic Therapy, RNA, Small Interfering, Gene Knockdown Techniques
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