Antisense Therapy for Cardiovascular Diseases

Title:Antisense Therapy for Cardiovascular Diseases

Volume: 21 Issue: 30

Author(s): M. Ian Phillips, Jessica Costales, Robert J. Lee, Edilamar Oliveira and Andrew B. Burns

Affiliation:

Keywords: Antisense oligonucleotides, metabolic syndrome, hypertension, hyperlipidemia, Kynamro™, Eteplirsen™, exon skipping.

Abstract: 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.

Cite this article as:

Phillips Ian M., Costales Jessica, Lee J. Robert, Oliveira Edilamar and Burns B. Andrew, Antisense Therapy for Cardiovascular Diseases, Current Pharmaceutical Design 2015; 21 (30) . https://dx.doi.org/10.2174/1381612821666150803150402