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用于神经退行性疾病研究的转基因非人灵长类动物。

Transgenic nonhuman primates for neurodegenerative diseases.

作者信息

Chan Anthony W S

机构信息

Yerkes National Primate Research Center and Department of Human Genetics, Emory University, Atlanta, Georgia, USA.

出版信息

Reprod Biol Endocrinol. 2004 Jun 16;2:39. doi: 10.1186/1477-7827-2-39.

Abstract

Animal models that represent human diseases constitute an important tool in understanding the pathogenesis of the diseases, and in developing effective therapies. Neurodegenerative diseases are complex disorders involving neuropathologic and psychiatric alterations. Although transgenic and knock-in mouse models of Alzheimer's disease, (AD), Parkinson's disease (PD) and Huntington's disease (HD) have been created, limited representation in clinical aspects has been recognized and the rodent models lack true neurodegeneration. Chemical induction of HD and PD in nonhuman primates (NHP) has been reported, however, the role of intrinsic genetic factors in the development of the diseases is indeterminable. Nonhuman primates closely parallel humans with regard to genetic, neuroanatomic, and cognitive/behavioral characteristics. Accordingly, the development of NHP models for neurodegenerative diseases holds greater promise for success in the discovery of diagnoses, treatments, and cures than approaches using other animal species. Therefore, a transgenic NHP carrying a mutant gene similar to that of patients will help to clarify our understanding of disease onset and progression. Additionally, monitoring disease onset and development in the transgenic NHP by high resolution brain imaging technology such as MRI, and behavioral and cognitive testing can all be carried out simultaneously in the NHP but not in other animal models. Moreover, because of the similarity in motor repertoire between NHPs and humans, it will also be possible to compare the neurologic syndrome observed in the NHP model to that in patients. Understanding the correlation between genetic defects and physiologic changes (e.g. oxidative damage) will lead to a better understanding of disease progression and the development of patient treatments, medications and preventive approaches for high risk individuals. The impact of the transgenic NHP model in understanding the role which genetic disorders play in the development of efficacious interventions and medications is foreseeable.

摘要

代表人类疾病的动物模型是理解疾病发病机制以及开发有效治疗方法的重要工具。神经退行性疾病是涉及神经病理和精神改变的复杂病症。尽管已经创建了阿尔茨海默病(AD)、帕金森病(PD)和亨廷顿舞蹈病(HD)的转基因和基因敲入小鼠模型,但已认识到其在临床方面的代表性有限,且啮齿动物模型缺乏真正的神经退行性变。已报道了在非人灵长类动物(NHP)中化学诱导HD和PD,然而,内在遗传因素在疾病发展中的作用尚不确定。非人灵长类动物在遗传、神经解剖以及认知/行为特征方面与人类极为相似。因此,与使用其他动物物种的方法相比,开发用于神经退行性疾病的NHP模型在发现诊断方法、治疗方法和治愈手段方面更有成功的希望。因此,携带与患者相似突变基因的转基因NHP将有助于阐明我们对疾病发作和进展的理解。此外,通过高分辨率脑成像技术(如MRI)以及行为和认知测试来监测转基因NHP中的疾病发作和发展,这些在NHP中都可以同时进行,但在其他动物模型中则无法做到。而且,由于NHP与人类在运动能力方面的相似性,也有可能将在NHP模型中观察到的神经综合征与患者的进行比较。了解遗传缺陷与生理变化(如氧化损伤)之间的相关性将有助于更好地理解疾病进展以及为患者开发治疗方法、药物和针对高危个体的预防措施。转基因NHP模型在理解遗传疾病在有效干预措施和药物开发中所起作用方面的影响是可预见的。

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