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脂质紊乱的新型生物技术治疗方法。

NEW BIOTECHNOLOGICAL TREATMENTS FOR LIPID DISORDERS.

作者信息

Valencia-Enciso Natalia, Mendivil Carlos O

机构信息

Department of Medicine, Universidad de los Andes School of Medicine, Bogotá, Colombia.

Department of Internal Medicine, Endocrinology Section, Fundación Santa Fe de Bogotá, Bogotá, Colombia.

出版信息

Rev Invest Clin. 2018;70(5):244-254. doi: 10.24875/RIC.18002577.

Abstract

Disorders of lipid and lipoprotein metabolism play a central role in the pathogenesis of atherosclerotic cardiovascular diseases (CVDs). Despite the widespread use of efficacious lipid-modifying therapies, the residual risk of CVD remains unacceptably high. The purpose of this manuscript is to review the application of new technologies in the treatment of lipid disorders. New therapies work mostly at the gene expression level and are, therefore, different from traditional small-molecule drugs that work mainly by inhibiting already synthesized proteins. We will briefly lay out the function of the gene products targeted by the new agents. Then, we will organize our review of new biotechnological treatments by the molecular approach, namely: monoclonal antibodies, antisense oligonucleotides, small-interfering RNAs, and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated 9 (Cas9)-based genome editing. The paper concludes with the description of the current clinical studies and the perspectives for the use of these agents. (REV INVEST CLIN. 2018;70:244-54).

摘要

脂质和脂蛋白代谢紊乱在动脉粥样硬化性心血管疾病(CVD)的发病机制中起着核心作用。尽管有效的脂质调节疗法已广泛应用,但CVD的残余风险仍然高得令人难以接受。本手稿的目的是综述新技术在脂质紊乱治疗中的应用。新疗法大多在基因表达水平起作用,因此不同于主要通过抑制已合成蛋白质起作用的传统小分子药物。我们将简要阐述新药物所靶向的基因产物的功能。然后,我们将按分子方法对新的生物技术治疗进行综述,即:单克隆抗体、反义寡核苷酸、小干扰RNA和基于成簇规律间隔短回文重复序列(CRISPR)/CRISPR相关蛋白9(Cas9)的基因组编辑。本文最后描述了当前的临床研究以及这些药物的使用前景。(《临床研究综述》。2018年;70:244 - 254)

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