Department of Psychiatry and Behavioral Sciences, Albert Einstein College of Medicine, New York, USA.
Department of Neuroscience, Albert Einstein College of Medicine, New York, USA.
Psychiatry Clin Neurosci. 2018 May;72(5):301-321. doi: 10.1111/pcn.12641. Epub 2018 Mar 1.
Copy number variants are deletions and duplications of a few thousand to million base pairs and are associated with extraordinarily high levels of autism spectrum disorder, schizophrenia, intellectual disability, or attention-deficit hyperactivity disorder. The unprecedented levels of robust and reproducible penetrance of copy number variants make them one of the most promising and reliable entry points to delve into the mechanistic bases of many mental disorders. However, the precise mechanistic bases of these associations still remain elusive in humans due to the many genes encoded in each copy number variant and the diverse associated phenotypic features. Genetically engineered mice have provided a technical means to ascertain precise genetic mechanisms of association between copy number variants and dimensional aspects of mental illnesses. Molecular, cellular, and neuronal phenotypes can be detected as potential mechanistic substrates for various behavioral constructs of mental illnesses. However, mouse models come with many technical pitfalls. Genetic background is not well controlled in many mouse models, leading to rather obvious interpretative issues. Dose alterations of many copy number variants and single genes within copy number variants result in some molecular, cellular, and neuronal phenotypes without a behavioral phenotype or with a behavioral phenotype opposite to what is seen in humans. In this review, I discuss technical and interpretative pitfalls of mouse models of copy number variants and highlight well-controlled studies to suggest potential neuronal mechanisms of dimensional aspects of mental illnesses. Mouse models of copy number variants represent toeholds to achieve a better understanding of the mechanistic bases of dimensions of neuropsychiatric disorders and thus for development of mechanism-based therapeutic options in humans.
拷贝数变异是几千到几百万个碱基对的缺失和重复,与自闭症谱系障碍、精神分裂症、智力障碍或注意力缺陷多动障碍的极高水平有关。拷贝数变异具有前所未有的稳健性和可重复性,使其成为深入研究许多精神障碍的机制基础的最有前途和最可靠的切入点之一。然而,由于每个拷贝数变异中编码了许多基因,并且与许多相关的表型特征有关,因此这些关联的精确机制基础在人类中仍然难以捉摸。基因工程小鼠为确定拷贝数变异与精神疾病维度之间的关联的精确遗传机制提供了技术手段。分子、细胞和神经元表型可以作为各种精神疾病的潜在机制底物进行检测。然而,小鼠模型存在许多技术陷阱。许多小鼠模型的遗传背景无法得到很好的控制,导致解释问题相当明显。许多拷贝数变异和拷贝数变异内的单个基因的剂量改变导致一些分子、细胞和神经元表型,而没有行为表型或具有与人类相反的行为表型。在这篇综述中,我讨论了拷贝数变异小鼠模型的技术和解释陷阱,并强调了控制良好的研究,以提出精神疾病维度的潜在神经元机制。拷贝数变异的小鼠模型代表了更好地理解神经精神障碍维度的机制基础的切入点,从而为人类开发基于机制的治疗选择提供了可能。