Swimming into prominence: the zebrafish as a valuable tool for studying human myopathies and muscular dystrophies.
Swimming into prominence: the zebrafish as a valuable tool for studying human myopathies and muscular dystrophies.
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DOI：
10.1111/febs.12412
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发表时间：
2013-09
期刊：
影响因子：
--
通讯作者：
Dowling JJ
中科院分区：
文献类型：
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作者：
Gibbs EM;Horstick EJ;Dowling JJ
肌肉疾病研究最近进入了一个令人兴奋的新阶段。下一代测序技术的应用推动了肌病和肌营养不良基因发现的前所未有的时代。针对杜氏肌营养不良症的基因疗法已进入临床试验，一些基于途径的疗法也正在针对少数肌肉疾病进行临床试验。虽然许多因素促进了基因发现和治疗开发的非凡发展，但斑马鱼模型系统已成为这些进步的重要工具。在这篇综述中，我们将重点介绍斑马鱼如何极大地帮助识别新的肌肉疾病基因和识别新的治疗策略。我们将从对斑马鱼作为模型的一般介绍开始，讨论在鱼中建模和分析肌肉疾病的方法，最后以最近研究的观察结果结束，这些研究强调了鱼作为肌肉疾病研究工具的力量。
A new and exciting phase of muscle disease research has recently been entered. The application of next generation sequencing technology has spurred an unprecedented era of gene discovery for both myopathies and muscular dystrophies. Gene‐based therapies for Duchenne muscular dystrophy have entered clinical trial, and several pathway‐based therapies are doing so as well for a handful of muscle diseases. While many factors have aided the extraordinary developments in gene discovery and therapy development, the zebrafish model system has emerged as a vital tool in these advancements. In this review, we will highlight how the zebrafish has greatly aided in the identification of new muscle disease genes and in the recognition of novel therapeutic strategies. We will start with a general introduction to the zebrafish as a model, discuss the ways in which muscle disease can be modeled and analyzed in the fish, and conclude with observations from recent studies that highlight the power of the fish as a research tool for muscle disease.
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