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From antibody to zinc finger: an A-Z of Open Targets

OpenTargets or Open Targets? Target validation or target identification? Celiac disease or coeliac disease? As users discover our resources, take a closer look at the lexicon of terms that matter to us.

This month we have not only one, but two new entries in our A-Z collection of blog posts. It is time for F and G.

Functional Genomics

Genomic.org.uk describes functional genomics as the “study of DNA with an aim to understand the relationship between the organism’s genome and phenotype”.

You may think there is nothing new to functional genomics, as gene-by-gene approaches of classical molecular biology techniques had exactly the same goal (Bunnik and Roch 2013). The novelty with functional genomics is its level of throughput and scale: a systematic and genome-wide investigation of the genotype-phenotype link.

Here at Open Targets, we focus on the relationship between human genomes and diseases through a portfolio of informatics and experimental projects. Our goal is to translate this understanding into safer and efficacious therapeutic drugs.

One of the techniques we have been employing towards this goal across our therapy areas is CRISPR-Cas9 screens. For instance, Fiona Behan and colleagues have perturbed >18,000 genes in 1,000 cancer cell lines, combined this with target tractability and genomic markers, to identify new drug targets, such as WRN for colorectal and ovarian carcinomas.


Open Targets is using CRISPR priority score to identify targets in cancer drug discovery. More Open Targets CRISPR data is on its way. Stay tuned!

The great news is that we will not be stopping at the target identification stage of drug discovery. This is just the start, albeit an important step.

We will soon launch the Open Targets Validation Lab and we are looking for a senior staff scientist to lead this outstanding endeavour. You will be based at the Wellcome Sanger Institute and lead the validation projects of our targets using robust and systematic experimental approaches.

Could this be you? Check the Sanger careers page for a detailed description of this role and how to apply.

Denise Carvalho-Silva

Denise Carvalho-Silva

Supporting drug discovery scientists, following post-doctorate research at the Australian National University and the Wellcome Sanger Institute, and working at Ensembl and GENCODE.

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