Division of Endocrinology, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
Department of Orthopaedic Surgery, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
Curr Osteoporos Rep. 2018 Aug;16(4):458-465. doi: 10.1007/s11914-018-0452-x.
The goal of this review is to highlight some of the considerations involved in creating animal models to study rare bone diseases and then to compare and contrast approaches to creating such models, focusing on the advantages and novel opportunities offered by the CRISPR-Cas system.
Gene editing after creation of double-stranded breaks in chromosomal DNA is increasingly being used to modify animal genomes. Multiple tools can be used to create such breaks, with the newest ones being based on the bacterial adaptive immune system known as CRISPR/Cas. Advances in gene editing have increased the ease and speed, while reducing the cost, of creating novel animal models of disease. Gene editing has also expanded the number of animal species in which genetic modification can be performed. These changes have significantly increased the options for investigators seeking to model rare bone diseases in animals.
本文旨在强调一些在创建用于研究罕见骨骼疾病的动物模型时需要考虑的问题,然后比较和对比创建这些模型的方法,重点介绍 CRISPR-Cas 系统提供的优势和新机会。
在染色体 DNA 双链断裂后进行基因编辑,越来越多地用于修饰动物基因组。有多种工具可用于创建此类断裂,最新的工具基于称为 CRISPR/Cas 的细菌适应性免疫系统。基因编辑技术的进步提高了创建疾病新型动物模型的简便性和速度,同时降低了成本。基因编辑还扩大了可以进行遗传修饰的动物物种数量。这些变化显著增加了研究人员在动物中模拟罕见骨骼疾病的选择。