/ Open Targets Platform

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.

Following antibody and breast carcinoma, it is time for our next letter, C.
No, it is not C for Christmas, but C for CRISPR/CAS9.

CRISPR

‘Crisper’, as it is pronounced, is many things. It is:

  • a genomic region in prokaryotes with short palindromic repeats, probably viral in origin
  • part of the adaptive immunity system of certain bacterial species to defend themselves against bacteriophage
  • “molecular scissors” to cut the genome in specific spots. The cuts are repaired by the cell when guide RNAs lead (hence their name) CAS9 to genes we want to engineer

crispr-cas9-1-1

CRISPR/CAS9 as molecular scissors. Image credit: What is CRISPR-Cas9?

In Open Targets, CRISPR/CAS9 is the work horse of many of our collaborative experimental projects. We use this editing tool to introduce, for example, loss-of-function changes in:

  • cancer cell lines to identify synthetic lethal interaction between cancer genomes and targeted gene deletions
  • human neurons to identify genes that regulate the efficiency of tau uptake
  • iPSC-derived neurons to identify genes that either confer resistance or sensitivity to oxidative stress, which has a role in neurodegenerative diseases

When should you be able to explore this data?

We hope these results will enable us to identify novel drug targets in some of the therapeutic areas we are interested in, such as Oncology and Neurodegeneration.

To be the first to know when these results are available in the Open Targets Platform, please subscribe to our Open Targets newsletter. Or email us if you have any questions.

In the meantime, merry CRISPR and a happy new year.

Dr Denise Carvalho-Silva

Dr Denise Carvalho-Silva

Scientific Outreach Lead at Open Targets after working in Ensembl, GENCODE, EUCOMM, pig genome projects, and post-doc at the Wellcome Sanger Institute and Australian National University.

Read More