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Action and Reaction: The Biological Response to siRNA and Its Delivery Vehicles
Open accessAbstract
RNA interference (RNAi)-based therapeutics have significant potential for the treatment of human disease. Safe and effective delivery of RNA to target tissues remains a major barrier to realizing its clinical potential. Several factors can affect the in vivo performance of short interfering RNA (siRNA) delivery formulations, including siRNA sequence, structure, chemical modification, and delivery formulation. This review provides an introduction to the principles underlying the pharmacokinetics and pharmacodynamics of systemically administered siRNA and its delivery formulations, including the factors that lead to its degradation, clearance, and tissue uptake, as well as its potential for immunogenicity, toxicity, and off-target effects within the body.
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Metadata
- Authors
- Kanasty, R. L., Whitehead, K. A., Vegas, A. J., & Anderson, D. G.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2012-03
- Volume
- 20
- Issue
- 3
- Pages
- 513-524
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2049233124
- Cited by
- 279
- References
- 170
- FWCI
- 10.57
- Type
- journal article
- Language
- en
- Open access
- hybrid
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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.