Delivery of RNA interference.
Over the last few years, RNA Interference (RNAi), a naturally occurring mechanism of gene regulation conserved in plant and mammalian cells, has opened numerous novel opportunities for basic research across the field of biology. While RNAi has helped accelerate discovery and understanding of gene functions, it also has great potential as a therapeutic and potentially prophylactic modality. Challenging diseases failing conventional therapeutics could become treatable by specific silencing of key pathogenic genes. More specifically, therapeutic targets previously deemed "undruggable" by small molecules, are now coming within reach of RNAi based therapy. For RNAi to be effective and elicit gene silencing response, the double-stranded RNA molecules must be delivered to the target cell. Unfortunately, delivery of these RNA duplexes has been challenging, halting rapid development of RNAi-based therapies. In this review we present current advancements in the field of siRNA delivery methods, including the pros and cons of each method.
Li
Charles X
CX
Division of Gastroenterology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts 02215, USA.
Parker
Amy
A
Menocal
Ellen
E
Xiang
Shuanglin
S
Borodyansky
Laura
L
Fruehauf
Johannes H
JH
D000818:Animals; D001419:Bacteria / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D005822:Genetic Vectors / Q000235:genetics / Q000627:therapeutic use; D006801:Humans; D008081:Liposomes / Q000627:therapeutic use; D049329:Nanostructures / Q000737:chemistry; D010957:Plasmids / Q000235:genetics; D034622:RNA Interference / Q000502:physiology*; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000235:genetics*; D014162:Transfection / Q000379:methods / Q000639:trends; D014780:Viruses / Q000235:genetics
D008081:Liposomes; D034741:RNA, Small Interfering