title: Antisense Therapy for Cardiovascular Diseases.
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.
authors.affiliation: Keck Graduate Institute, Claremont, CA, USA. ian_phillips@kgi.edu.
authors.forename: M Ian
authors.initials: MI
authors.lastname: Phillips
authors.forename: Jessica
authors.initials: J
authors.lastname: Costales
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Lee
authors.forename: Edilamar
authors.initials: E
authors.lastname: Oliveira
authors.forename: Andrew B
authors.initials: AB
authors.lastname: Burns
mesh_terms: D002318:Cardiovascular Diseases / Q000188:drug therapy* / Q000235:genetics*; D005091:Exons; D006801:Humans; D009154:Mutation; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*
chemical_list: D016376:Oligonucleotides, Antisense