期刊
CURRENT PHARMACEUTICAL DESIGN
卷 21, 期 30, 页码 4417-4426出版社
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1381612821666150803150402
关键词
Antisense oligonucleotides; metabolic syndrome; hypertension; hyperlipidemia; Kynamro (TM); Eteplirsen (TM); exon skipping
资金
- [FAPESP/SPRINT 2014/50673-4]
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 (TM) (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.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据