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作为研究人类神经退行性疾病的模型系统。

as a Model System to Study Human Neurodegenerative Disorders.

机构信息

Department of Physiology, Medical School, National and Kapodistrian University of Athens, GR-157 27 Athens, Greece.

Department of Biology, University of Padova, 35100 Padova, Italy.

出版信息

Biomolecules. 2023 Mar 5;13(3):478. doi: 10.3390/biom13030478.

Abstract

In recent years, advances in science and technology have improved our quality of life, enabling us to tackle diseases and increase human life expectancy. However, longevity is accompanied by an accretion in the frequency of age-related neurodegenerative diseases, creating a growing burden, with pervasive social impact for human societies. The cost of managing such chronic disorders and the lack of effective treatments highlight the need to decipher their molecular and genetic underpinnings, in order to discover new therapeutic targets. In this effort, the nematode serves as a powerful tool to recapitulate several disease-related phenotypes and provides a highly malleable genetic model that allows the implementation of multidisciplinary approaches, in addition to large-scale genetic and pharmacological screens. Its anatomical transparency allows the use of co-expressed fluorescent proteins to track the progress of neurodegeneration. Moreover, the functional conservation of neuronal processes, along with the high homology between nematode and human genomes, render extremely suitable for the study of human neurodegenerative disorders. This review describes nematode models used to study neurodegeneration and underscores their contribution in the effort to dissect the molecular basis of human diseases and identify novel gene targets with therapeutic potential.

摘要

近年来,科学技术的进步提高了我们的生活质量,使我们能够攻克疾病并延长人类的预期寿命。然而,长寿伴随着与年龄相关的神经退行性疾病频率的增加,给人类社会带来了日益沉重的负担和广泛的社会影响。管理这类慢性疾病的成本以及缺乏有效的治疗方法凸显出需要解析其分子和遗传基础,以便发现新的治疗靶点。在这方面,线虫是一个强大的工具,可以重现多种与疾病相关的表型,并提供了一个高度可塑的遗传模型,允许实施多学科方法,以及大规模的遗传和药理学筛选。它的解剖透明性允许使用共表达的荧光蛋白来跟踪神经退行性的进展。此外,神经元过程的功能保守性,以及线虫和人类基因组之间的高度同源性,使得线虫非常适合研究人类神经退行性疾病。本文描述了用于研究神经退行性变的线虫模型,并强调了它们在解析人类疾病的分子基础和识别具有治疗潜力的新基因靶点方面的贡献。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/01db/10046667/17410ead2646/biomolecules-13-00478-g001.jpg

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