RNAi works in monkeys

Apparent absence of toxicity and persistence of silencing may bode well for humans

Charles Choi
Mar 26, 2006
A disease-causing gene was silenced in monkeys through RNA interference (RNAi) therapy delivered into the bloodstream, scientists reported online yesterday (March 26) in Nature. This is the first study to show that systemic administration of RNAi works in non-human primates, and the findings affirm the promise of this new type of therapy, scientists say.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 of the City of Hope in Duarte, Calif., who did not participate in this study, told The Scientist. 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),...

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