斑马鱼中与建模相关的肌萎缩侧索硬化症
Modelling -Related Amyotrophic Lateral Sclerosis in Zebrafish.
作者信息
Fortier Gabrielle, Butti Zoé, Patten Shunmoogum A
机构信息
INRS-Centre Armand-Frappier Santé et Biotechnologie, Laval, QC H7V 1B7, Canada.
Centre d'Excellence en Recherche sur les Maladies Orphelines-Fondation Courtois (CERMO-FC), Université du Québec à Montréal (UQAM), Montréal, QC H2X 3Y7, Canada.
出版信息
Biomedicines. 2020 Oct 21;8(10):440. doi: 10.3390/biomedicines8100440.
Abstract
A hexanucleotide repeat expansion within the gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and its discovery has revolutionized our understanding of this devastating disease. Model systems are a valuable tool for studying ALS pathobiology and potential therapies. The zebrafish () has particularly become a useful model organism to study neurological diseases, including ALS, due to high genetic and physiological homology to mammals, and sensitivity to various genetic and pharmacological manipulations. In this review we summarize the zebrafish models that have been used to study the pathology of -related ALS. We discuss their value in providing mechanistic insights and their potential use for drug discovery.
摘要
该基因内的六核苷酸重复扩增是肌萎缩侧索硬化症（ALS）最常见的遗传病因，其发现彻底改变了我们对这种毁灭性疾病的认识。模型系统是研究ALS病理生物学和潜在治疗方法的宝贵工具。斑马鱼由于与哺乳动物具有高度的遗传和生理同源性，以及对各种遗传和药物操作的敏感性，尤其成为研究包括ALS在内的神经疾病的有用模式生物。在本综述中，我们总结了用于研究与该基因相关的ALS病理学的斑马鱼模型。我们讨论了它们在提供机制见解方面的价值及其在药物发现中的潜在用途。
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