微缩模型:用黑腹果蝇研究人类神经退行性变

Q3 Pharmacology, Toxicology and Pharmaceutics Drug Discovery Today: Disease Models Pub Date : 2017-12-01 DOI:10.1016/j.ddmod.2018.09.004
Roald Lambrechts , Anita Faber , Ody Sibon
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引用次数: 5

摘要

尽管在临床诊断、遗传学和分子生物学方面取得了巨大进步,但神经退行性疾病,如帕金森病(PD)、阿尔茨海默病(AD)和亨廷顿病(HD),无论是在对其病理生理的理解还是在治疗方面,仍然面临着巨大的挑战。能够充分模拟疾病机制的复杂性并对潜在治疗作出反应的生物体,同时不妥协于易于处理,研究和操作以研究它们,代表了转化生物学和医学的圣杯。在这里,我们回顾了果蝇黑腹果蝇作为神经变性领域的模式生物的适用性。我们简要地总结了这一有机体的科学研究历史,回顾了现有的分子、遗传和药理学工具箱,并讨论了这些工具箱在神经退行性疾病研究中的应用方法。最后,通过回顾果蝇的一些发现,这些发现随后被转化为其他生物体并在其走向临床的过程中得到验证,强调了黑腹果蝇的力量和稳健性。
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Modelling in miniature: Using Drosophila melanogaster to study human neurodegeneration

Despite great advances in clinical diagnostics, genetics and molecular biology, neurodegenerative diseases like Parkinson’s disease (PD), Alzheimer’s disease (AD) and Huntington’s disease (HD) still pose great challenges, both in terms of understanding their pathophysiology as well as their treatment. Organisms able to adequately model the intricacies of the disease mechanism and respose to potential treatment, whilst not compromising on ease of handling, studying and manipulating in order to study them, represent the holy grail of translational biology and medicine. Here, we review the suitability of the fruit fly, Drosophila melanogaster, as a model organism in the field of neurodegeneration. We briefly summarize the history of scientific research concerning this organism, review the molecular, genetic and pharmacological toolbox available and we discuss the ways this toolbox has been applied to research in neurodegeneration. Finally, by reviewing some findings in the fruit fly which were subsequently translated to and validated in other organisms on their way to the clinic, the power and robustness of Drosophila melanogaster is highlighted.

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来源期刊
Drug Discovery Today: Disease Models
Drug Discovery Today: Disease Models Pharmacology, Toxicology and Pharmaceutics-Drug Discovery
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期刊介绍: Drug Discovery Today: Disease Models discusses the non-human experimental models through which inference is drawn regarding the molecular aetiology and pathogenesis of human disease. It provides critical analysis and evaluation of which models can genuinely inform the research community about the direct process of human disease, those which may have value in basic toxicology, and those which are simply designed for effective expression and raw characterisation.
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