Standardization of zebrafish drug testing parameters for muscle diseases.

Standardization of zebrafish drug testing parameters for muscle diseases.
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DOI:
10.1242/dmm.050339
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发表时间:
2024-01-01
影响因子:
4.3
通讯作者:
--
中科院分区:
医学2区
文献类型:
--
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骨骼肌疾病主要影响骨骼肌和心肌,导致肌肉无力、呼吸功能受损和寿命缩短。这些有害的结果导致健康相关的生活质量差,并带来很高的医疗保健经济负担。由于缺乏有前途的治疗方法和新的肌肉疾病疗法,需要新的方法来识别候选药物和在动物模型中的进步。因此,在模拟人类肌肉疾病特征的动物模型中快速筛选药物化合物是必要的。斑马鱼是临床前研究中的多功能模型,支持开发生物学和药物发现计划,用于新的化学实体和重新利用已建立的药物。由于几个优点,有越来越多的应用程序的斑马鱼模型的高通量药物筛选人类疾病和发育研究。因此,关键药物筛选参数的标准化,如畜牧业协议,药物化合物的管理和结果的措施,是至关重要的模型和领域的持续发展。在这里,我们试图总结和探索关键的药物治疗和药物筛选参数在斑马鱼为基础的建模人类肌肉疾病。通过改进药物筛选参数和方案的标准化和协调化,我们的目标是促进更有效的药物发现计划。总结:我们提出了一个标准化的药物筛选和评价标准,在斑马鱼肌肉疾病模型,以提高实验的严谨性,可重复性和翻译的目标。
Skeletal muscular diseases predominantly affect skeletal and cardiac muscle, resulting in muscle weakness, impaired respiratory function and decreased lifespan. These harmful outcomes lead to poor health-related quality of life and carry a high healthcare economic burden. The absence of promising treatments and new therapies for muscular disorders requires new methods for candidate drug identification and advancement in animal models. Consequently, the rapid screening of drug compounds in an animal model that mimics features of human muscle disease is warranted. Zebrafish are a versatile model in preclinical studies that support developmental biology and drug discovery programs for novel chemical entities and repurposing of established drugs. Due to several advantages, there is an increasing number of applications of the zebrafish model for high-throughput drug screening for human disorders and developmental studies. Consequently, standardization of key drug screening parameters, such as animal husbandry protocols, drug compound administration and outcome measures, is paramount for the continued advancement of the model and field. Here, we seek to summarize and explore critical drug treatment and drug screening parameters in the zebrafish-based modeling of human muscle diseases. Through improved standardization and harmonization of drug screening parameters and protocols, we aim to promote more effective drug discovery programs. Summary: We propose a standardization of criteria for drug screening and evaluation in zebrafish muscle disease models, with the goal of improving experimental rigor, reproducibility and translation.
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