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ACS Chem Biol. 2013 Jul 19;8(7):1402-6. doi: 10.1021/cb4001316. Epub 2013 May 2.

Lipid nanoparticles improve activity of single-stranded siRNA and gapmer antisense oligonucleotides in animals.

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  • 1Department of Medicinal Chemistry, Isis Pharmaceuticals, Inc. , 1896 Rutherford Road, Carlsbad, California 92008, United States.


We evaluated the abilities of an antisense oligonucleotide (ASO), a small interfering RNA (siRNA), and a single-stranded siRNA (ss-siRNA) to inhibit expression from the PTEN gene in mice when formulated identically with lipid nanoparticles (LNPs). Significantly greater reductions in levels of PTEN mRNA were observed for LNP-formulated agents compared to unformulated drugs when gene silencing was evaluated after a single dose in the livers of mice. An unformulated ss-siRNA modified with a metabolically stable phosphate mimic 5'-(E)-vinylphosphonate showed dose-dependent reduction of PTEN mRNA in mice, albeit at doses significantly higher than those observed for formulated ss-siRNA. These results demonstrate that LNPs can be used to deliver functional antisense and ss-siRNA therapeutics to the liver, indicating that progress in the field of siRNA delivery is transferable to other classes of nucleic acid-based drugs.

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