功能基因组学
什么是功能基因组学?
功能基因组学 is a rapidly advancing drug discovery method that can be used to explore links between our genes and their effects. 使用功能基因组学, we can identify and investigate genes and biological processes that are associated with diseases and which can be explored as potential drug targets.
基因组编辑是功能基因组学的关键工具, making it possible to delete or change genes in cells to understand their roles in disease. 最著名的基因组编辑工具之一是CRISPR.
功能基因组学中的基因组编辑技术
许多技术可用于研究功能基因组学. But by far the most effective and versatile is the revolutionary gene editing technology CRISPR/Cas9 – or CRISPR (Clustered Regularly Interspaced Short Palindromic Repeat). 2015年，它被命名为 年度突破 by 科学 杂志已迅速成为一种广泛而有价值的研究工具.
CRISPR represents a breakthrough approach to editing genes and many variants have been developed. 在最简单的应用中, CRISPR acts as molecular scissors that can be used to precisely cut and modify a DNA sequence of interest. 准确的, 可编程和适应性强, this technology has found widespread application across several areas of biological and biopharmaceutical research.
澳门葡京赌博游戏是最早投资CRISPR的制药公司之一, 2014年在哥德堡和剑桥成立了专门的研究小组. The ability to ‘edit’ genes using CRISPR may be one of the most significant discoveries in the history of biology. It not only has potential as a tool to study and understand disease in functional genomics, but in the future even has potential to treat disease with a genetic cause.
功能基因组学的最新进展
基因与疾病的联系
The primary reason that candidate drugs fail in clinical trials is because they do not have a significant effect on the course of disease.
Selecting the right target is therefore the single most important decision we make in the drug discovery process.
寻找正确的目标
功能基因组学 helps us to identify the right targets for drug discovery, uncovering genes and biological processes that can be targeted to help slow, 阻止或逆转疾病.
By using functional genomics to guide drug discovery we can deliver precision medicines that target the underlying causes of disease.
By targeting every single gene in the genome and understanding the networks in which they function, hand-in-hand with novel treatment approaches such as antisense oligonucleotides, 澳门葡京赌博游戏可以扩大治疗的范围.
功能基因组学是如何应用的?
We are exploring several ways that functional genomics can be used to support drug discovery. 越来越多地, we are able to perform large-scale CRISPR screens that integrate data science and artificial intelligence (AI) to help uncover meaningful patterns in the data.
基因淘汰赛
检查从基因组中移除一个基因的效果. Widely used to identify genes that when deleted lead to resistance to cancer medicines.
疾病模型
通过操纵细胞或动物模型中的基因来模拟疾病状态, 允许测试新药的效果. 比以前的方法快得多.
高通量筛选
Combining biology and computing to analyse large amounts of CRISPR data to understand ‘big picture’ links between genes and disease using machine learning and AI.
The key to maximising the chances of success in target identification lies in the pairing of the latest genome editing technologies with bioinformatics and AI to efficiently analyse the data generated from screenings.
你能帮助澳门葡京赌博游戏找到更多改变生活的药物吗? 申请加入团队.
AstraZeneca supports me to develop my skills with some of the latest technologies, and to work closely with a wide range of leading experts in genome editing.
合作发现功能基因组学的新疗法
人类功能基因组学计划
Together with the Medical Research Council and the Milner Therapeutics Institute, 澳门葡京赌博游戏正在创建一个功能基因组学实验室, 能够进行大规模的CRISPR筛选. 该设施支持 澳门葡京赌博游戏的野心 改变慢性病患者的护理方式.
This unique collaboration enables open sharing of resources and expertise across academia and industry, and plays a focal role in the UK-wide 人类功能基因组学计划. 该实验室支持哮喘等疾病的发现研究, 心血管, 代谢性和炎症性疾病.
功能基因组学中心与癌症研究视野
专攻肿瘤学, the AstraZeneca–Cancer Research Horizons Functional Genomics Centre (FGC) is a world-leader in genetic screening, 癌症模型, CRISPR试剂设计和大数据处理的计算方法. We are developing CRISPR technology to better understand the biology of cancer and creating pre-clinical models which are more reflective of human disease.
威康桑格研究所
Since 2015, we have collaborated with the 威康桑格研究所 on CRISPR technology. 这一合作于2018年延长, giving us access to its leading libraries of gRNA for silencing or activating every gene in the genome via CRISPR.
通过共同努力，澳门葡京赌博游戏可以做得更快. Our functional genomics partnerships are revealing more insights into disease biology and allowing shared access to the latest technologies and leading expertise.
