In the study, gene silencing using the highest dose persisted for 11 days, without any observed toxicity, a finding that "bodes well for future human systemic trials of RNAi therapies," John Rossi of the Beckman Research Institute.
Scientists at Alnylam Pharmaceuticals and Protiva Biotherapeutics gave cynomolgus monkeys a single injection of small interfering RNAs (siRNAs) against the gene for apolipoprotein B (ApoB). This gene is involved in the assembly and secretion of low-density lipoprotein (LDL), and has not proven accessible to targeting with conventional small molecule or protein therapies.
Gene silencing occurred in a dose-dependent manner. Within 48 hours, the maximum level of APOB silencing exceeded 90%, with a more than 75% reduction in plasma ApoB and a more than 80% drop in LDL.
A report on other RNAi related work
RNAi therapy has been proposed by Ray Kurzweil and Bill Joy as part of the basis for a broad program of defence against natural and artificial viruses.