Antisense oligonucleotide therapy is a growing field in cardiac, metabolic, and muscular diseases. This
precision therapy allows for treatment of diseases due to specific genetic defects. Antisense has few side effects
and is relatively long lasting. Some major targets for antisense therapy include hyperglycemia, hyperlipidemia, and
hypercholesterolemia. ISIS Pharmaceuticals recently commercialized antisense therapy with Kynamro™
(Mipomersen) for homozygous familial hypercholesterolemia, opening the door for other antisense oligonucleotides
for lowering proteins. Antisense can also be used to increase proteins that are inhibited by mutant exons.
Sarepta is testing exon 51 skipping in the mutated dystrophin gene, which if successful will help affected individuals
walk, and may help restore some cardiac function. These antisense techniques also could be applied as antisense
therapies to overcome gene defects in hypertension, heart disease, muscular defects and metabolic syndrome.
Keywords: Antisense oligonucleotides, metabolic syndrome, hypertension, hyperlipidemia, Kynamro™, Eteplirsen™, exon skipping.
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