Ma Dongyuan, Liu Feng
State Key Laboratory of Membrane Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China.
State Key Laboratory of Membrane Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China.
Genomics Proteomics Bioinformatics. 2015 Dec;13(6):336-44. doi: 10.1016/j.gpb.2015.12.001. Epub 2016 Jan 4.
Technological advances are important for innovative biological research. Development of molecular tools for DNA manipulation, such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the clustered regularly-interspaced short palindromic repeat (CRISPR)/CRISPR-associated (Cas), has revolutionized genome editing. These approaches can be used to develop potential therapeutic strategies to effectively treat heritable diseases. In the last few years, substantial progress has been made in CRISPR/Cas technology, including technical improvements and wide application in many model systems. This review describes recent advancements in genome editing with a particular focus on CRISPR/Cas, covering the underlying principles, technological optimization, and its application in zebrafish and other model organisms, disease modeling, and gene therapy used for personalized medicine.
技术进步对于创新性生物学研究至关重要。用于DNA操作的分子工具的发展,如锌指核酸酶(ZFNs)、转录激活样效应物核酸酶(TALENs)以及成簇规律间隔短回文重复序列(CRISPR)/CRISPR相关蛋白(Cas),已经彻底改变了基因组编辑。这些方法可用于开发潜在的治疗策略以有效治疗遗传性疾病。在过去几年中,CRISPR/Cas技术取得了重大进展,包括技术改进以及在许多模型系统中的广泛应用。本综述描述了基因组编辑的最新进展,特别关注CRISPR/Cas,涵盖其基本原理、技术优化及其在斑马鱼和其他模型生物中的应用、疾病建模以及用于个性化医疗的基因治疗。