RNA-interference effectors and their delivery.
RNA-interference, the mechanism by which the expression of a specific protein can be reduced or eliminated, has emerged as a potential way to treat disease. RNA-interference effectors, such as small interfering RNA and small hairpin RNA, are double-stranded nucleic acid sequences expressly designed to have homology to sections of the target protein's mRNA, and when present in the cytosol trigger hydrolysis of the mRNA through the RNA-interference pathway. Because RNA-interference effectors are macromolecular and typically polyanionic, their efficacy is poor when not accompanied by a delivery vehicle. This review outlines the RNA-interference mechanism and discusses the delivery of RNA-interference effectors, with an emphasis on synthetic polymer-based delivery systems.
Putnam
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Department of Biomedical Engineering and the School of Chemical and Biomolecular Engineering, Cornell University, Ithaca, NY, USA.
Doody
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D000818:Animals; D001943:Breast Neoplasms / Q000628:therapy; D003505:Cyclodextrins; D016503:Drug Delivery Systems / Q000379:methods; D018274:Electroporation; D005260:Female; D005822:Genetic Vectors; D006801:Humans; D011094:Polyethyleneimine / Q000737:chemistry; D011479:Protamines; D034622:RNA Interference / Q000502:physiology*; D020871:RNA Stability; D034741:RNA, Small Interfering / Q000502:physiology*
D003505:Cyclodextrins; D011479:Protamines; D034741:RNA, Small Interfering; D011094:Polyethyleneimine