Journal
ADVANCED DRUG DELIVERY REVIEWS
Volume 61, Issue 9, Pages 760-766Publisher
ELSEVIER
DOI: 10.1016/j.addr.2009.04.006
Keywords
Short hairpin RNA; In vivo delivery; Duration; Side effect; Hydrodynamic delivery
Categories
Funding
- Ministry of Education, Science, Sports, and Culture of Japan
- Ministry of Health, Labour and Welfare of Japan
- National Institute of Biomedical Innovation (NIBIO)
- Grants-in-Aid for Scientific Research [21390009] Funding Source: KAKEN
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RNA interference (RNAi) is a potent and specific gene silencing event in which small interfering RNA (siRNA) degrades target mRNA. Therefore, RNAi is of potential use as a therapeutic approach for the treatment of a variety of diseases in which aberrant expression of mRNA causes a problem. RNAi can be achieved by delivering siRNA or vectors that transcribe siRNA or short-hairpin RNA (shRNA). The aim of this review is to examine the potential of nonviral vector-mediated RNAi technology in treating diseases. The characteristics of plasmid DNA expressing shRNA were compared with those of siRNA, focusing on the duration of gene silencing, delivery to target cells and target specificity. Recent progresses in prolonging the RNAi effect, improving the delivery to target cells and increasing the specificity of RNAi in vivo are also reviewed. (C) 2009 Elsevier B.V. All rights reserved.
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