Advances in lipid nanoparticles for siRNA delivery
Open accessAbstract
Technological advances in both siRNA (small interfering RNA) and whole genome sequencing have demonstrated great potential in translating genetic information into siRNA-based drugs to halt the synthesis of most disease-causing proteins. Despite its powerful promises as a drug, siRNA requires a sophisticated delivery vehicle because of its rapid degradation in the circulation, inefficient accumulation in target tissues and inability to cross cell membranes to access the cytoplasm where it functions. Lipid nanoparticle (LNP) containing ionizable amino lipids is the leading delivery technology for siRNA, with five products in clinical trials and more in the pipeline. Here, we focus on the technological advances behind these potent systems for siRNA-mediated gene silencing.
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Appropriate delivery methods are essential for enabling systemic applications of RNA therapeutics and maximizing their therapeutic window.
“Although some naked RNAs and chemically modified RNAs were applied for therapies based on local administration or ex vivo applications, appropriate delivery methods are still essential for both enabling systemic applications of RNA therapeutics and further maximizing their therapeutic window.[22]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [22]
Different non-viral materials have been designed and optimized for accommodating efficient delivery of diverse RNA molecules as well as multi-component genome editing systems.
“Different non-viral materials have been designed and optimized for accommodating efficient delivery of diverse RNA molecules as well as multi-component genome editing systems.[22, 34–36]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [22]
LNPs represent a major intravenously administered siRNA delivery system for clinical applications.
“Among the reported non-viral siRNA delivery platforms, LNPs represent a major intravenously (i.v.) administered siRNA delivery system for clinical applications.[22, 38]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [22]
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- Authors
- Tam, Y., Chen, S., & Cullis, P.
- Affiliations
- University of British Columbia (CA)
- Corresponding author
- Tam, Yuen
- Venue
- Pharmaceutics
- Publisher
- MDPI AG
- Published
- 2013-09-18
- Volume
- 5
- Issue
- 3
- Pages
- 498-507
- ISSN
- 1999-4923 (ISSN-L)
- PMC
- PMC3836621
- OpenAlex
- W2034139519
- Cited by
- 229
- References
- 61
- Citations by year
- 2026: 15 · 2025: 17 · 2024: 22 · 2023: 24 · 2022: 19 · 2021: 19 · 2020: 19 · 2019: 24 · 2018: 17 · 2017: 10 · 2016: 18 · 2015: 13 · 2014: 11 · 2013: 1
- FWCI
- 6.42
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Pharmaceutics (publishedVersion, cc-by)
- Europe PMC (PubMed Central) (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- cIRcle (University of British Columbia) (submittedVersion, cc-by-nc-sa)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Open Collections
- Funders
- Alnylam Pharmaceuticals
- Canadian Institutes of Health Research
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advancements in Transdermal Drug Delivery
- Keywords
- lipid nanoparticles, siRNA delivery, gene silencing, siRNA degradation, clinical trials, RNA interference therapy
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