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- Title
Action and Reaction: The Biological Response to siRNA and Its Delivery Vehicles.
- Authors
Kanasty, Rosemary L; Whitehead, Kathryn A; Vegas, Arturo J; Anderson, Daniel G
- Abstract
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.
- Subjects
RNA interference; CELL membranes; CHEMICAL modification of proteins; PHARMACODYNAMICS; TOXICITY testing
- Publication
Molecular Therapy, 2012, Vol 20, Issue 3, p513
- ISSN
1525-0016
- Publication type
Article
- DOI
10.1038/mt.2011.294