疾病
转基因小鼠
转基因
表型
神经科学
生物
遗传模型
医学
动物模型
病态的
计算生物学
条件基因敲除
基因剔除小鼠
人类疾病
模式生物
体内
基因工程
临床表型
遗传异质性
复杂疾病
人体病理学
生物信息学
遗传性疾病
变性(医学)
脊椎动物
遗传学
人类蛋白质
内生
治疗方法
基因敲除
作者
Valina L. Dawson,Ted M. Dawson
标识
DOI:10.1101/cshperspect.a041644
摘要
Parkinson's disease (PD) is a complex genetic disorder that is associated with environmental risk factors and aging. Vertebrate genetic models, especially in mice, has aided the study of autosomal-dominant and autosomal-recessive PD. Mice are capable of exhibiting a broad range of phenotypes and coupled with their conserved genetic and anatomical structures provides unparalleled molecular and pathological tool to model human disease. These models used in combination with aging and PD-associated toxins have expanded our understanding of PD pathogenesis. Attempts to refine PD animal models using conditional approaches have yielded in vivo nigrostriatal degeneration that is instructive in ordering pathogenic signaling and in developing therapeutic strategies to cure or halt the disease. α-Synuclein preformed fibril (PFF) injections, which induce the aggregation of endogenous α-synuclein, remarkably recapitulate pathological processes observed in human PD. Here, we provide an overview of the generation and characterization of transgenic and knockout mice and the α-synuclein PFF models used to study PD followed by molecular insights that have been gleamed these PD mouse models.
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