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Animal models for researching approaches to therapy of Duchenne muscular dystrophy
Transgenic Research ( IF 3 ) Pub Date : 2021-08-18 , DOI: 10.1007/s11248-021-00278-3
M I Zaynitdinova 1 , A V Lavrov 1 , S A Smirnikhina 1
Affiliation  

Duchenne muscular dystrophy (DMD) is a relatively widespread genetic disease which develops as a result of a mutation in the gene DMD encoding dystrophin. In this review, animal models of DMD are described. These models are used in preclinical studies to elucidate the pathogenesis of the disease or to develop effective treatments; each animal model has its own advantages and disadvantages. For instance, Caenorhabditis elegans, Drosophila melanogaster, and zebrafish (sapje) are suitable for large-scale chemical screening of large numbers of small molecules, but their disease phenotype differs from that of mammals. The use of larger animals is important for understanding of the potential efficacy of various treatments for DMD. While mdx mice have their advantages, they exhibit a milder disease phenotype compared to humans or dogs, making it difficult to evaluate the efficacy of new treatment for DMD. The disease in dogs and pigs is more severe and progresses faster than in mice, but it is more difficult to breed and obtain sufficient numbers of specimens in order to achieve statistically significant results. Moreover, working with large animals is also more labor-intensive. Therefore, when choosing the optimal animal model for research, it is worth considering all the goals and objectives.



中文翻译:

研究杜氏肌营养不良症治疗方法的动物模型

杜氏肌营养不良症 (DMD) 是一种相对普遍的遗传疾病,它是由于编码肌营养不良蛋白的基因DMD发生突变而发展起来的。在这篇综述中,描述了 DMD 的动物模型。这些模型用于临床前研究,以阐明疾病的发病机制或开发有效的治疗方法;每种动物模型都有自己的优点和缺点。例如,秀丽隐杆线虫黑腹果蝇斑马鱼sapje) 适用于大量小分子的大规模化学筛选,但其疾病表型与哺乳动物不同。使用较大的动物对于了解各种治疗 DMD 的潜在功效很重要。虽然 mdx 小鼠有其优势,但与人类或狗相比,它们表现出更温和的疾病表型,因此难以评估新疗法对 DMD 的疗效。与小鼠相比,狗和猪的疾病更严重,进展更快,但更难以繁殖和获得足够数量的标本以达到统计学上显着的结果。此外,与大型动物一起工作也更加劳动密集。因此,在选择用于研究的最佳动物模型时,值得考虑所有目标和目的。

更新日期:2021-08-19
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