Antisense Therapy for Cardiovascular Diseases

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

Journal Name: Current Pharmaceutical Design

Volume 21 , Issue 30 , 2015

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.

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

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Article Details

VOLUME: 21
ISSUE: 30
Year: 2015
Page: [4417 - 4426]
Pages: 10
DOI: 10.2174/1381612821666150803150402
Price: $58

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