Functional genomics

通过CRISPR基因编辑来理解疾病并使药物发现成为可能


What is functional genomics?

功能基因组学是一种快速发展的药物发现方法,可用于探索澳门第一赌城在线娱乐的基因及其作用之间的联系. Using functional genomics, 澳门第一赌城在线娱乐可以识别和研究与疾病相关的基因和生物过程,并将其作为潜在的药物靶点进行探索.

Genome editing is a key tool in functional genomics, 使删除或改变细胞中的基因成为可能,从而了解它们在疾病中的作用. One of the most well-known genome editing tools is CRISPR.




Genome editing technologies in functional genomics


了解基因编辑技术以及CRISPR如何增加澳门第一赌城在线娱乐对疾病的理解.

A number of technologies are available to study functional genomics. 但到目前为止,最有效和最通用的是革命性的基因编辑技术CRISPR/Cas9 -或CRISPR(聚集规则间隔短回文重复). In 2015 it was named the breakthrough of the year by Science magazine and has rapidly become a widespread and much valued research tool.

CRISPR代表了一种突破性的基因编辑方法,许多变体已经被开发出来. In its simplest application, CRISPR就像分子剪刀,可以用来精确地剪切和修改感兴趣的DNA序列. Accurate, programmable and adaptable, 这项技术已被广泛应用于生物和澳门第一赌城在线娱乐研究的几个领域.

We were one of the first pharmaceutical companies to invest in CRISPR, setting up dedicated research groups in Gothenburg and Cambridge in 2014. 使用CRISPR“编辑”基因的能力可能是生物学历史上最重要的发现之一. 它不仅有潜力作为研究和理解功能基因组学疾病的工具, 但在未来,它甚至有可能治疗由基因引起的疾病.
 








通过瞄准基因组中的每一个基因并了解它们发挥作用的网络, 与新的治疗方法如反义寡核苷酸携手并进, we can expand the therapeutic world that is available to us.

Steve Rees SVP, Discovery Sciences, BioPharmaceuticals R&D, AstraZeneca



How is functional genomics being used?

澳门第一赌城在线娱乐正在探索几种功能基因组学可以用来支持药物发现的方法. Increasingly, 澳门第一赌城在线娱乐能够进行大规模的CRISPR筛选,将数据科学和人工智能(AI)结合起来,帮助发现数据中有意义的模式.



将最新的基因组编辑技术与生物信息学和人工智能相结合,有效地分析筛选产生的数据,是最大限度地提高目标识别成功率的关键.

Davide Gianni Senior Director, Functional Genomics, Discovery Sciences, R&D, AstraZeneca



Can you help us find more life-changing medicines? Apply to join the team.


澳门在线赌城娱乐支持我用一些最新的技术来发展我的技能, and to work closely with a wide range of leading experts in genome editing.





Collaborating to discover new therapies with functional genomics

Human Functional Genomics Initiative

还有医学研究委员会和米尔纳治疗研究所, we are creating a functional genomics laboratory, capable of large-scale CRISPR screening. The facility supports our BioPharmaceuticals ambition of transforming care for people with chronic diseases.

这种独特的合作使学术界和工业界能够开放共享资源和专业知识, 并在英国范围内的人类功能基因组计划中发挥着重要作用. The lab supports discovery research in diseases such as asthma, cardiovascular, metabolic and inflammatory diseases.

Functional Genomics Centre with Cancer Research Horizons

Specialising in oncology, 澳门在线赌城娱乐癌症研究视野功能基因组学中心(FGC)是全球领先的基因筛查中心, cancer models, CRISPR reagent design and computational approaches to big data processing. 澳门第一赌城在线娱乐正在开发CRISPR技术,以更好地了解癌症的生物学,并创建更能反映人类疾病的临床前模型.

Wellcome Sanger Institute

自2015年以来,澳门第一赌城在线娱乐与Wellcome Sanger Institute合作研究CRISPR技术. This collaboration was extended in 2018, 让澳门第一赌城在线娱乐可以访问其领先的gRNA文库,通过CRISPR沉默或激活基因组中的每个基因.

We can do more faster by working together. 澳门第一赌城在线娱乐的功能基因组合作伙伴关系正在揭示更多关于疾病生物学的见解,并允许共享最新技术和领先专业知识.





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Veeva ID: Z4-57937
Date of preparation: September 2023