Antisense Oligonucleotides in Dyslipidemia Management: A Review of Clinical Trials.

Abstract

Introduction: Elevated serum total cholesterol levels, very low-density lipoprotein cholesterol, low-density lipoprotein cholesterol, triglycerides, or a decreased serum high-density lipoprotein cholesterol concentration characterize dyslipidemia. Antisense Oligonucleotide therapy in dyslipidemia targets apolipoprotein B (ApoB), an essential component of low-density lipoprotein (LDL) associated with atherosclerosis development.

Aim: This review aims to critically evaluate the efficacy and safety of this group of medications in mitigating dyslipidemia in at-risk individuals and its potential role in advancing personalized medicine in the management of dyslipidemias.

Methods: A detailed search was conducted from multiple databases adhering to the PRISMA guidelines. Clinical trials and randomized controlled trials on antisense oligonucleotides for management of dyslipidemias were included, excluding non-English studies, case reports and all forms of reviews. Data was screened, with duplicates removed, and key findings were synthesized using a narrative approach.

Results and conclusion: The potential of antisense oligonucleotides (ASOs) to treat dyslipidemia and other disorders has attracted much interest. Several studies and clinical trials have been conducted on the safety and tolerability of ASOs for dyslipidemia. Although statins are the mainstay management of hypercholesterolemia, there is evidence from clinical trials that ASOs can even be more effective with little to no side effects. Novel therapeutic approaches such as antisense oligonucleotides (ASOs) offer tailored therapeutic alternatives. ASOs such as Mipomersen and Volanesorsen provide additional treatment options for patients with inherited lipid abnormalities by lowering certain atherogenic lipoproteins such as apo B and ApoC-III, respectively.