Department of Medicine, Baylor College of Medicine, Michael E. DeBakey Veterans Affairs Medical Center, One Baylor Plaza, Houston, TX, 77030, USA,
Curr Atheroscler Rep. 2015 Jan;17(1):467. doi: 10.1007/s11883-014-0467-4.
Antisense oligonucleotide therapy is a promising approach for the treatment of a broad variety of medical conditions. It functions at the cellular level by interfering with RNA function, often leading to degradation of specifically targeted abnormal gene products implicated in the disease process. Mipomersen is a novel antisense oligonucleotide directed at apolipoprotein (apoB)-100, the primary apolipoprotein associated with low-density lipoprotein cholesterol (LDL-C), which has recently been approved for the treatment of familial hypercholesterolemia. A number of clinical studies have demonstrated its efficacy in lowering LDL-C and apoB levels in patients with elevated LDL-C despite maximal medical therapy using conventional lipid-lowering agents. This review outlines the risks and benefits of therapy and provides recommendations on the use of mipomersen.
反义寡核苷酸疗法是一种有前途的治疗方法，可用于治疗多种医学病症。它通过干扰 RNA 功能在细胞水平上发挥作用，通常导致特异性靶向异常基因产物的降解，这些基因产物与疾病过程有关。米泊美生是一种新型的针对载脂蛋白 (apoB)-100 的反义寡核苷酸，apoB-100 是与低密度脂蛋白胆固醇 (LDL-C) 相关的主要载脂蛋白，最近已被批准用于治疗家族性高胆固醇血症。许多临床研究表明，尽管使用常规降脂药物进行最大程度的药物治疗，但该药在降低 LDL-C 和 apoB 水平方面具有疗效。本文概述了该疗法的风险和益处，并就米泊美生的使用提供了建议。
