Antisense oligonucleotides in the treatment of lipid disorders: pitfalls and promises.

Dyslipidaemia is one of the pivotal risk factors for cardiovascular disease (CVD), and lipid-lowering therapy is therefore the cornerstone in cardiovascular risk management. With the currently available treatment options the relative risk reduction in CVD is approximately 30%, leaving a large residual risk. This calls for the development of additional therapeutic moieties and antisense oligonucleotides (ASOs) have proven to be such a new and effective treatment. ASOs are short single strands of DNA that intracellularly bind mRNA of specific proteins. This induces the degradation of the mRNA through which the protein cannot be produced. Based on knowledge of lipid metabolism several targets of ASO therapy can be identified. This review offers a summary of current developments in ASO therapy regarding lipid disorders.