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In Vivo Delivery of RNAi with Lipid-Based Nanoparticles
Abstract
RNA interference (RNAi) technology represents a fundamentally new category of treatments for human disease by addressing targets that are traditionally considered undruggable with existing medicines. The major challenge for RNAi-based therapy is the delivery system that meets human therapeutic needs. Therefore, engineering vectors with good delivery efficiency and safety profile is an intense area of research. Lipid-based nanoparticles for RNAi have yielded successful advances in vivo and to an extent in clinical trials. In this review, we discuss the barriers in developing lipid-based nanoparticles for in vivo RNAi and different strategies to overcome them. Rational designs that address safety concerns and ensure effective delivery will aid the translation of engineered lipid-based nanoparticles toward the clinic in the foreseeable future.
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“The exceptional properties of LNPs for RNA delivery include, but are not limited to, the encapsulation and protection of RNAs from degradation during circulation, as well as the delivery of RNA payloads to the target organ following systemic administration.[18, 54, 61]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [61]
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Metadata
- Authors
- Huang, L., & Liu, Y.
- Affiliations
- University of North Carolina at Chapel Hill (US)
- ORCID
- Huang, Leaf
- Venue
- Annual Review of Biomedical Engineering
- Publisher
- Annual Reviews
- Published
- 2011-08-15
- Volume
- 13
- Issue
- 1
- Pages
- 507-530
- ISSN
- 1523-9829 (ISSN-L)
- 1545-4274
- OpenAlex
- W2127833150
- Cited by
- 174
- References
- 115
- Citations by year
- 2026: 5 · 2025: 2 · 2024: 5 · 2023: 6 · 2022: 8 · 2021: 9 · 2020: 8 · 2019: 10 · 2018: 9 · 2017: 12 · 2016: 17 · 2015: 8 · 2014: 18 · 2013: 34 · 2012: 21
- FWCI
- 2.18
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Annual Review of Biomedical Engineering (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institutes of Health
- National Cancer Institute (CA129421, R01CA129835, R01CA129421, R01CA149363, CA149363, CA129835)
- 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 › Advanced Drug Delivery Systems
- Keywords
- RNA interference, lipid nanoparticles, gene silencing, therapeutic vectors, vector design, vector safety, clinical translation, undruggable targets
- MeSH terms
- Algorithms, Drug Carriers, Genetic Vectors, Humans, Liposomes, Neoplasms, Pharmacokinetics, Polyethylene Glycols, Virus Diseases, Genetic Therapy, Genetic Diseases, Inborn, RNA Interference, RNA, Small Interfering, Nanoparticles
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