Gene editing in common cardiovascular diseases

IF 12 1区 医学 Q1 PHARMACOLOGY & PHARMACY
Anna-Maria Lauerer , Xurde M. Caravia , Lars S. Maier , Francesco Chemello , Simon Lebek
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引用次数: 0

Abstract

Cardiovascular diseases are the leading cause of morbidity and mortality worldwide, highlighting the high socioeconomic impact. Current treatment strategies like compound-based drugs or surgeries are often limited. On the one hand, systemic administration of substances is frequently associated with adverse side effects; on the other hand, they typically provide only short-time effects requiring daily intake. Thus, new therapeutic approaches and concepts are urgently needed. The advent of CRISPR-Cas9 genome editing offers great promise for the correction of disease-causing hereditary mutations. As such mutations are often very rare, gene editing strategies to correct them are not broadly applicable to many patients. Notably, there is recent evidence that gene editing technology can also be deployed to disrupt common pathogenic signaling cascades in a targeted, specific, and efficient manner, which offers a more generalizable approach. However, several challenges remain to be addressed ranging from the optimization of the editing strategy itself to a suitable delivery strategy up to potential immune responses to the editing components. This review article discusses important CRISPR-Cas9-based gene editing approaches with their advantages and drawbacks and outlines opportunities in their application for treatment of cardiovascular diseases.

常见心血管疾病的基因编辑
心血管疾病是全球发病率和死亡率的主要原因,对社会经济影响巨大。目前的治疗策略,如复方药物或手术,往往存在局限性。一方面,全身用药经常会产生不良副作用;另一方面,这些药物通常只能在短期内见效,需要每天服用。因此,迫切需要新的治疗方法和理念。CRISPR-Cas9 基因组编辑技术的出现为纠正致病遗传突变带来了巨大希望。由于这类基因突变通常非常罕见,因此纠正这些突变的基因编辑策略并不能广泛适用于许多患者。值得注意的是,最近有证据表明,基因编辑技术也可用于以靶向、特异和高效的方式破坏常见的致病信号级联,这提供了一种更具普遍性的方法。然而,从编辑策略本身的优化到合适的传递策略,再到对编辑成分的潜在免疫反应,仍有一些挑战有待解决。这篇综述文章讨论了基于CRISPR-Cas9的重要基因编辑方法及其优缺点,并概述了这些方法在心血管疾病治疗中的应用机会。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
23.00
自引率
0.70%
发文量
222
审稿时长
90 days
期刊介绍: Pharmacology & Therapeutics, in its 20th year, delivers lucid, critical, and authoritative reviews on current pharmacological topics.Articles, commissioned by the editor, follow specific author instructions.This journal maintains its scientific excellence and ranks among the top 10 most cited journals in pharmacology.
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