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Inhibiting recycling pathways to enhance cellular retention of lipid nanoparticle (LNP)-delivered small interfering RNAs

Inhibiting exocytosis of LNP-delivered siRNAs from endosomes could enhance their retention within the cell. In cultured cells, knockout of Niemann-Pick disease type C1 (NPC1), which encodes a protein important for endosome recycling, increased siRNA accumulation and gene silencing compared with no knockout. In cells, genetic depletion of other endosome recycling targets yielded comparable outcomes. Next steps could include targeting proteins and lipids involved in biogenesis and endosome trafficking to enhance cellular retention of LNP-delivered siRNA.

SciBX 6(30); doi:10.1038/scibx.2013.808
Published online Aug. 8, 2013

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Sahay, G. et al. Nat. Biotechnol.; published online June 23, 2013;
Contact: Daniel G. Anderson, Massachusetts Institute of Technology, Cambridge, Mass.