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Delivery materials for siRNA therapeutics
Abstract
RNA interference (RNAi) has broad potential as a therapeutic to reversibly silence any gene. To achieve the clinical potential of RNAi, delivery materials are required to transport short interfering RNA (siRNA) to the site of action in the cells of target tissues. This Review provides an introduction to the biological challenges that siRNA delivery materials aim to overcome, as well as a discussion of the way that the most effective and clinically advanced classes of siRNA delivery systems, including lipid nanoparticles and siRNA conjugates, are designed to surmount these challenges. The systems that we discuss are diverse in their approaches to the delivery problem, and provide valuable insight to guide the design of future siRNA delivery materials.
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“Several miRNAs and antisense oligonucleotides (ASOs) are currently under investigation in clinical trials for various diseases (Kanasty et al., 2013;van Rooij and Kauppinen, 2014;Gaudet et al., 2015).”
From microRNA-33 Regulates ApoE Lipidation and Amyloid-β Metabolism in the Brain (2015) · cites this paper as Kanasty et al., 2013
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“The main role of PEGylated lipid in LNPs is to reduce aggregation and non-specific uptake by immune cells.304–306”
From Advances in COVID-19 mRNA vaccine development (2022) · cites this paper as [306]
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“we believe that recent advances in the delivery of nucleic acid therapeutics provide hope that CRISPR-Cas9–mediated correction may be translatable to humans15.”
From Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype (2014) · cites this paper as [15]
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Metadata
- Authors
- Kanasty, R., Dorkin, J. R., Vegas, A., & Anderson, D.
- Affiliations
- Koch Institute for Integrative Cancer Research At MIT (US)
- Boston Children's Hospital (US)
- Corresponding author
- Anderson, Daniel
- Venue
- Nature Materials
- Published
- 2013-10-23
- Volume
- 12
- Issue
- 11
- Pages
- 967-977
- ISSN
- 1476-1122 (ISSN-L)
- 1476-4660
- OpenAlex
- W1995770815
- Cited by
- 1,800
- References
- 113
- Citations by year
- 2026: 77 · 2025: 103 · 2024: 111 · 2023: 138 · 2022: 139 · 2021: 148 · 2020: 159 · 2019: 129 · 2018: 164 · 2017: 151 · 2016: 183 · 2015: 179 · 2014: 114 · 2013: 4
- FWCI
- 15.04
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Nature Materials (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Physical Sciences › Materials Science › Polymers and Plastics › Dendrimers and Hyperbranched Polymers
- Keywords
- siRNA delivery, RNA interference therapy, lipid nanoparticles, siRNA conjugates, gene silencing, drug delivery materials
- MeSH terms
- Animals, Drug Carriers, Humans, RNA, Small Interfering, Nanoparticles
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