Recent advances using zebrafish animal models for muscle disease drug discovery.

Recent advances using zebrafish animal models for muscle disease drug discovery.
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DOI:
10.1517/17460441.2014.927435
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发表时间:
2014-09
影响因子:
6.3
通讯作者:
Maves L
Maves L
中科院分区:
医学2区
文献类型:
--
作者:
Maves L

文献摘要

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动物模型使发现和理解治疗肌肉疾病如杜氏肌营养不良症的药理学方法取得了巨大进展。通过这篇文章,作者为读者提供了斑马鱼动物模型的描述,该模型已被用于识别和研究肌肉疾病的药理学方法。特别是,作者重点介绍了大规模化学筛选和靶向药物治疗研究如何将斑马鱼确立为肌肉疾病药物发现的重要模型。利用斑马鱼模型进一步开发肌肉疾病的药理学方法有许多机会,包括研究药物联合疗法和利用基因组编辑来设计斑马鱼肌肉疾病模型。作者特别认为,广泛的斑马鱼转基因品系用于标记免疫细胞类型的可用性,结合肌肉疾病模型的实时成像和药物治疗,应该允许新的优雅研究,证明药理学方法如何影响肌肉疾病中的炎症和免疫反应。
Animal models have enabled great progress in the discovery and understanding of pharmacological approaches for treating muscle diseases like Duchenne muscular dystrophy. With this article, the author provides the reader with a description of the zebrafish animal model, which has been employed to identify and study pharmacological approaches to muscle disease. In particular, the author focuses on how both large-scale chemical screens and targeted drug treatment studies have established zebrafish as an important model for muscle disease drug discovery. There are a number of opportunities arising for the use of zebrafish models for further developing pharmacological approaches to muscle diseases, including studying drug combination therapies and utilizing genome editing to engineer zebrafish muscle disease models. It is the author’s particular belief that the availability of a wide range of zebrafish transgenic strains for labeling immune cell types, combined with live imaging and drug treatment of muscle disease models, should allow for new elegant studies demonstrating how pharmacological approaches might influence inflammation and the immune response in muscle disease.