achieving efficient rnai therapy: progress and challenges

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2013
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Abstract
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.
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Authors ;Kun Gao;Leaf Huang
Journal energy policy
Year 2013
DOI
10.1016/j.apsb.2013.06.005
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