Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
2474647 | Acta Pharmaceutica Sinica B | 2013 | 13 Pages |
RNA interference (RNAi) has been harnessed to produce a new class of drugs for treatment of various diseases. This review summarizes the most important parameters that govern the silencing efficiency and duration of the RNAi effect such as small interfering RNA (siRNA) stability and modification, the type of delivery system and particle sizing methods. It also discusses the predominant barriers for siRNA delivery, such as off-target effects and introduces internalization, endosomal escape and mathematical modeling in RNAi therapy and combinatorial RNAi. At present, effective delivery of RNAi therapeutics in vivo remains a challenge although significant progress has been made in this field.
Graphical abstractSignificant progress has been made in effective delivery of RNAi therapeutics in vivo, although it remains a challenge in this field. This review summarizes the most important parameters that govern the silencing efficiency and duration of the RNAi effect, such as small interfering RNA (siRNA) stability and modification, the type of delivery system and particle sizing methods. It also discusses the predominant barriers for siRNA delivery, such as off-target effects and introduces internalization, endosomal escape and mathematical modeling in RNAi therapy and combinatorial RNAi.Figure optionsDownload full-size imageDownload as PowerPoint slide