Barrangou Rodolphe, Doudna Jennifer A
Department of Food, Bioprocessing and Nutrition Sciences, North Carolina State University, Raleigh, North Carolina, USA.
Howard Hughes Medical Institute, Innovative Genomics Initiative, Department of Molecular and Cell Biology, University of California, Berkeley, California, USA.
Nat Biotechnol. 2016;34(9):933-941. doi: 10.1038/nbt.3659. Epub 2016 Sep 8.
Programmable DNA cleavage using CRISPR-Cas9 enables efficient, site-specific genome engineering in single cells and whole organisms. In the research arena, versatile CRISPR-enabled genome editing has been used in various ways, such as controlling transcription, modifying epigenomes, conducting genome-wide screens and imaging chromosomes. CRISPR systems are already being used to alleviate genetic disorders in animals and are likely to be employed soon in the clinic to treat human diseases of the eye and blood. Two clinical trials using CRISPR-Cas9 for targeted cancer therapies have been approved in China and the United States. Beyond biomedical applications, these tools are now being used to expedite crop and livestock breeding, engineer new antimicrobials and control disease-carrying insects with gene drives.
使用CRISPR-Cas9进行可编程DNA切割可在单细胞和整个生物体中实现高效、位点特异性的基因组工程。在研究领域,多功能的基于CRISPR的基因组编辑已被广泛应用于多种方式,如控制转录、修饰表观基因组、进行全基因组筛选和染色体成像。CRISPR系统已被用于减轻动物的遗传疾病,并且可能很快会应用于临床治疗人类眼部和血液疾病。在中国和美国,两项使用CRISPR-Cas9进行靶向癌症治疗的临床试验已获批准。除了生物医学应用,这些工具现在还被用于加速作物和牲畜育种、设计新型抗菌药物以及利用基因驱动控制病媒昆虫。