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7/22/2011 PERMALINK
RNA-based cholesterol drug is readied for human tests.
Alnylam Pharmaceuticals has filed a clinical trial application to test a new RNA-based treatment on 32 U.K. volunteers. The RNA targets an enzyme involved in clearing cholesterol from the blood. A single dose has already been shown to reduce LDL, or 'bad,' cholesterol, in nonhuman primates by as much as 50 percent. The technique co-opts a method of gene silencing employed by our own cells. Using short lengths of small interfering RNA (siRNA) to interrupt gene transcription and prevent the production of proteins produced by the gene. The process promises to allow the regulation of troublesome genes that cause diseases and cellular dysfunction due to aging.