用于心血管疾病研究的基因组编辑
Genome Editing for the Study of Cardiovascular Diseases.
作者信息
Chadwick Alexandra C, Musunuru Kiran
机构信息
Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
出版信息
Curr Cardiol Rep. 2017 Mar;19(3):22. doi: 10.1007/s11886-017-0830-5.
PURPOSE OF REVIEW
The opportunities afforded through the recent advent of genome-editing technologies have allowed investigators to more easily study a number of diseases. The advantages and limitations of the most prominent genome-editing technologies are described in this review, along with potential applications specifically focused on cardiovascular diseases.
RECENT FINDINGS
The recent genome-editing tools using programmable nucleases, such as zinc-finger nucleases, transcription activator-like effector nucleases, and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9), have rapidly been adapted to manipulate genes in a variety of cellular and animal models. A number of recent cardiovascular disease-related publications report cases in which specific mutations are introduced into disease models for functional characterization and for testing of therapeutic strategies. Recent advances in genome-editing technologies offer new approaches to understand and treat diseases. Here, we discuss genome editing strategies to easily characterize naturally occurring mutations and offer strategies with potential clinical relevance.
综述目的
近期基因组编辑技术的出现带来了诸多机遇，使研究人员能够更轻松地研究多种疾病。本综述将介绍最主要的基因组编辑技术的优缺点，以及专门针对心血管疾病的潜在应用。
最新发现
近期使用可编程核酸酶的基因组编辑工具，如锌指核酸酶、转录激活样效应因子核酸酶和规律成簇的间隔短回文重复序列（CRISPR）/CRISPR相关蛋白9（Cas9），已迅速应用于在多种细胞和动物模型中操纵基因。近期一些与心血管疾病相关的出版物报道了将特定突变引入疾病模型以进行功能表征和治疗策略测试的案例。基因组编辑技术的最新进展为理解和治疗疾病提供了新方法。在此，我们讨论基因组编辑策略，以轻松表征自然发生的突变，并提供具有潜在临床相关性的策略。
Zotero 插件