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OVERVIEW OF CURRENT MOUSE MODELS OF AUTISM AND STRATEGIES FOR THEIR DEVELOPMENT USING CRISPR/Cas9 TECHNOLOGY

机译:使用CRISPR / CAS9技术概述自闭症和策略的自闭症和战略策略

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摘要

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 anddescribes 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:自闭症谱系疾病(ASD)是一组高度异质神经疾病,被认为具有强遗传组分。由于人体医学中功能基因组学方法有限,为复杂人类疾病的研究创造了足够的动物模型表现出极大的潜力。有几种已经成熟的闭锁模型是对单一表型特征的洞察力,尽管其复杂表型的原因尚未完全理解。开发新技术,如CRISPR / CAS9,代表了目标基因组工程和建立的巨大能力新动物模型。本文提供了最新的概述了自闭症基因组学领域的当前知识概述,涉及用于建立新的小鼠模型的CRISPR / CAS9技术的潜力,代表SGRNA设计作为规划CRISPR / CAS9单敲击的最初步骤之一-out实验。此外,它还概述了当前的行为研究方法,说明如何开发相关的动物模型。

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