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Knocking down barriers: advances in siRNA delivery | Nature Reviews Drug Discovery
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RNA interference holds vast potential as a therapeutic strategy for both disease prevention and treatment, but its use has so far been hampered by a lack of safe and effective delivery techniques. In their Review, Anderson and colleagues discuss the challenges associated with small interfering RNA delivery and highlight promising novel synthetic delivery agents. 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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permissions reprints peg-ylated magnetic-fluid-loaded liposomes nature portfolio privacy policy acid-labile Ξ²-thiopropionate linkage ph-responsive metal-organic framework advertising integrated cancer research lipid-mediated dna-transfection procedure rna-induced silencing complex nuclease-resistant double-stranded rna social media nobel prize-winning discovery research tool nanoparticle research low-ph-sensitive poly combinatorial library age-related macular degeneration author information authors anti-inflammatory host factor exploiting delivery system-induced rnai-mediated gene silencing rnai-mediated gene-targeting sirna-mediated gene silencing nature cell biol l-domain-dependent manner author correspondence vitamin a-coupled liposomes block copolymer/sirna polyplexes target-independent angiogenesis suppression sequence-specific gene silencing small interfering rnas polyelectrolyte complex micelles position-specific chemical modification large library bcr-abl1+ leukemia cells sequence-specific potent induction polyethylenimine-mediated sirna delivery vivo anti-hbv activity small interfering rna springerlink instant access live-animal bioluminescent imaging enhance sirna activity nucleic acid delivery permissions nature rev nature biotech nature mater short interfering rna nature med
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RNA interference holds vast potential as a therapeutic strategy for both disease prevention and treatment, but its use has so far been hampered by a lack of safe and effective delivery techniques. In their Review, Anderson and colleagues discuss the challenges associated with small interfering RNA delivery and highlight promising novel synthetic delivery agents. 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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