Alves-Bezerra Michele, Furey Nika, Johnson Collin G, Bissig Karl-Dimiter
Center for Cell and Gene Therapy, Stem Cells and Regenerative Medicine Center, Baylor College of Medicine, Houston, TX, USA.
Stem Cells and Regenerative Medicine Center (STAR), Baylor College of Medicine, Houston, TX, USA.
JHEP Rep. 2019 Oct 25;1(5):392-402. doi: 10.1016/j.jhepr.2019.09.002. eCollection 2019 Nov.
CRISPR/Cas9 gene editing has revolutionised biomedical research. The ease of design has allowed many groups to apply this technology for disease modelling in animals. While the mouse remains the most commonly used organism for embryonic editing, CRISPR is now increasingly performed with high efficiency in other species. The liver is also amenable to somatic genome editing, and some delivery methods already allow for efficient editing in the whole liver. In this review, we describe CRISPR-edited animals developed for modelling a broad range of human liver disorders, such as acquired and inherited hepatic metabolic diseases and liver cancers. CRISPR has greatly expanded the repertoire of animal models available for the study of human liver disease, advancing our understanding of their pathophysiology and providing new opportunities to develop novel therapeutic approaches.
CRISPR/Cas9基因编辑彻底改变了生物医学研究。其设计简便,使得许多研究团队能够将这项技术应用于动物疾病建模。虽然小鼠仍是胚胎编辑最常用的生物体,但CRISPR现在在其他物种中也越来越高效地进行。肝脏也适合进行体细胞基因组编辑,一些递送方法已经能够在整个肝脏中进行高效编辑。在本综述中,我们描述了为模拟多种人类肝脏疾病而开发的CRISPR编辑动物,如获得性和遗传性肝脏代谢疾病以及肝癌。CRISPR极大地扩展了可用于研究人类肝脏疾病的动物模型库,增进了我们对其病理生理学的理解,并为开发新的治疗方法提供了新机会。