Small interfering RNAs (siRNAs) can be systemically administered in non-human primates and significantly reduce expression from the mRNA against which they are directed. A therapeutic effect can also be achieved by targeting a microRNA (miRNA). A modified oligonucleotide directed against miR-122 was administered to monkeys, resulting in a reduction in serum cholesterol without detectable toxicity.
- Joacim Elmén
- Morten Lindow
- Sakari Kauppinen
