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. 2015 Mar 6;347(6226):1069-70.
doi: 10.1126/science.1252967.

RNA interference. Drugging RNAi

Dirk Haussecker  1 , Mark A Kay  2
Affiliations

RNA interference. Drugging RNAi

Dirk Haussecker et al. Science. .
No abstract available

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Figures

Figure
Figure. RNAi is the drug
RNAi delivery approaches include conjugates, liposomal nanoparticles (LNPs), and viral vectors. Nanoparticles deliver synthetic dsRNA, whereas viral vectors deliver a transcriptional template to the nucleus. Cellular uptake occurs nonspecifically or via receptor-mediated endocytosis. Nanoparticles deliver the RNAi trigger to the cytoplasm. The trigger enters the RNAi pathway at the RISC or Dicer processing stage. Transcriptional templates produce hairpin RNAs that enter the pathway at an earlier, nuclear stage. Ultimately, an active RISC complex is formed that cleaves the mRNA target. RISC, RNA-induced silencing complex; shRNA, short hairpin-mediated RNA.
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