Center for Stem Cell and Regenerative Disease, Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases (IMM), University of Texas-Health Science Center at Houston, Houston, Texas, 77030.
J Cell Biochem. 2017 Dec;118(12):4152-4162. doi: 10.1002/jcb.26111. Epub 2017 May 31.
The revolutionary technology for genome editing known as the clustered regularly interspaced short palindromic repeat (CRISPR)-CRISPR-associated protein 9 (Cas9) system has sparked advancements in biological and biomedical research. The scientific breakthrough of the development of CRISPR-Cas9 technology has allowed us to recapitulate human diseases by generating animal models of interest ranging from zebrafish to non-human primates. The CRISPR-Cas9 system can also be used to delineate the mechanisms underlying the development of human disorders and to precisely correct disease-causing mutations. Repurposing this technology enables wider applications in transcriptome and epigenome manipulation and holds promise to reach the clinic. In this review, we highlight the latest advances of the CRISPR-Cas9 system in different platforms and discuss the hurdles and challenges this technology is facing. J. Cell. Biochem. 118: 4152-4162, 2017. © 2017 Wiley Periodicals, Inc.
被称为成簇规律间隔短回文重复（CRISPR）-CRISPR 相关蛋白 9（Cas9）系统的革命性基因编辑技术在生物和生物医学研究领域引发了进步。CRISPR-Cas9 技术的发展是一项科学突破，使我们能够通过生成从斑马鱼到非人类灵长类动物的各种感兴趣的动物模型来重现人类疾病。CRISPR-Cas9 系统还可用于阐明人类疾病发展的机制，并精确纠正致病突变。该技术的重新应用使转录组和表观基因组操纵的应用更加广泛，并有望进入临床。在这篇综述中，我们强调了 CRISPR-Cas9 系统在不同平台上的最新进展，并讨论了该技术面临的障碍和挑战。细胞生化学杂志 118:4152-4162，2017。© 2017 威利父子公司
