Loading...
Antisense oligonucleotides are RNA molecules designed to selectively bind mRNA, which codes for specific proteins. An antisense oligonucleotide that inhibits apolipoprotein B100 was approved by the FDA in 2013 for treatment of homozygous familial hypercholesterolemia. Now, researchers report results of manufacturer-sponsored, randomized, double-blind, placebo-controlled, dose-ranging studies of two other antisense oligonucleotides targeting different lipid risk factors.
Gaudet and colleagues tested ISIS 304801, an antisense oligonucleotide directed against apolipoprotein C-III (APOC3), a molecule associated with very-low-density lipoproteins that inhibits lipoprotein lipase and hepatic lipase, thereby inducing hypertriglyceridemia. Elevated …