Abstract
Antisense technology might be a gateway to the treatment of diseases by targeting the expression of genes rather than permanently altering them. Thus, there will be fewer ethical concerns. Antisense oligonucleotides (ASO) can alter target gene expression by binding to RNA. Once bound, the ASO either disables or induces the degradation of the target RNA. This technology may be used to treat various conditions (including cancer, diabetes, and hypertension, as well as autoimmune and cardiovascular diseases). ASOs are potentially potent, selective and well-tolerated drugs. Mipomersen (ISIS 301012) inhibits human apolipoprotein (apo)B-100 synthesis and lowers circulating apoB and low-density lipoprotein cholesterol levels. ASO technology may provide a spectrum of agents targeting other vascular risk factors or mediators of atherosclerosis.
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