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Overview of current mouse models of autism and strategies for their development using CRISPR/Cas9 technology

机译:当前自闭症小鼠模型概述及其使用CRISPR / Cas9技术的发展策略

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Autism spectrum disorders (ASD) are a group of highly heterogenous neurological disorders that are believed to have strong genetic component. Due to the limited use of approaches of functional genomics in human medicine, creating adequate animal models for the study of complex human diseases shows great potential. There are several already established mouse models of autism that offer insight into single phenotypic traits, although causes for its complex phenotype have not yet been fully understood. Development of new technologies, such as CRISPR/Cas9, represent great capability for targeted genome engineering and establishment of new animal models. This article provides an up to date overview of current knowledge in the area of autism genomics and describes the potential of CRISPR/Cas9 technology for the establishment of new mouse models, representing sgRNA design as one of the initial steps in planning a CRISPR/Cas9 single knock-out experiment. In addition, it offers an overview of current approaches to behavioural studies, explaining how relevant animal models could be developed.
机译:自闭症谱系障碍(ASD)是一组高度异质的神经系统疾病,据信具有强大的遗传成分。由于人类医学中功能基因组学方法的使用有限,因此为研究复杂的人类疾病创建适当的动物模型显示出巨大的潜力。尽管尚未完全了解其复杂表型的成因,但已有几种已经建立的自闭症小鼠模型可以洞察单个表型特征。 CRISPR / Cas9等新技术的开发代表了靶向基因组工程和建立新动物模型的强大能力。本文提供了自闭症基因组学领域最新知识的最新概述,并介绍了CRISPR / Cas9技术在建立新小鼠模型中的潜力,代表sgRNA设计是计划CRISPR / Cas9单株的初始步骤之一淘汰赛实验。此外,它概述了当前的行为研究方法,解释了如何开发相关的动物模型。

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