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Knocking down barriers: advances in siRNA delivery
Open accessAbstract
In the 10 years that have passed since the Nobel prize-winning discovery of RNA interference (RNAi), billions of dollars have been invested in the therapeutic application of gene silencing in humans. Today, there are promising data from ongoing clinical trials for the treatment of age-related macular degeneration and respiratory syncytial virus. Despite these early successes, however, the widespread use of RNAi therapeutics for disease prevention and treatment requires the development of clinically suitable, safe and effective drug delivery vehicles. Here, we provide an update on the progress of RNAi therapeutics and highlight novel synthetic materials for the encapsulation and intracellular delivery of nucleic acids.
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“Recently, there has been an exponential growth in the development of clinically suitable nonviral delivery systems for siRNA (19,20); we have taken advantage of these innovations to develop the LNP/RNA formulation and have now embarked on a more extensive evaluation of other nonviral delivery systems.”
From Nonviral delivery of self-amplifying RNA vaccines (2012) · cites this paper as [20]
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“There are limited published data on the in vivo delivery of self-amplifying RNA using nonviral delivery strategies (3,14,18), and none has taken advantage of the recently developed, clinically suitable delivery systems for siRNA (19,20).”
From Nonviral delivery of self-amplifying RNA vaccines (2012) · cites this paper as [20]
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“Additionally, endosomal membranes have been damaged or rebuilt using cell penetrating peptides to enhance the intracellular delivery of nucleic acids.313”
From Smart nanoparticles for cancer therapy (2023) · cites this paper as [313]
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Metadata
- Authors
- Whitehead, K. A., Langer, R., & Anderson, D. G.
- Affiliations
- Massachusetts Institute of Technology (US)
- Koch Institute for Integrative Cancer Research At MIT (US)
- Corresponding author
- Anderson, Daniel G.
- Venue
- Nature Reviews Drug Discovery
- Published
- 2009-02
- Volume
- 8
- Issue
- 2
- Pages
- 129-138
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- PMC
- PMC7097568
- OpenAlex
- W1964146045
- Cited by
- 3,115
- References
- 106
- Citations by year
- 2026: 96 · 2025: 114 · 2024: 150 · 2023: 120 · 2022: 159 · 2021: 207 · 2020: 148 · 2019: 191 · 2018: 183 · 2017: 199 · 2016: 220 · 2015: 218 · 2014: 240 · 2013: 233 · 2012: 222
- FWCI
- 16.16
- Type
- journal article
- Language
- en
- Update notices
- Open access
- bronze
- doi.org
- Licence
- Locations
- Nature Reviews Drug Discovery (publishedVersion)
- PubMed Central (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
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
- Keywords
- siRNA delivery, RNA interference therapy, gene silencing, nucleic acid encapsulation, intracellular delivery, age-related macular degeneration, respiratory syncytial virus, clinical trials
- MeSH terms
- Animals, Clinical Trials as Topic, Humans, Liposomes, Drug Delivery Systems, RNA Interference, RNA, Small Interfering, Nanoparticles
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