Question: How do you use genetic data to find new drugs?

Reka Nagy answered on 8 Jan 2019:

I realised I messed up my one-sentence description! I actually use genetics to find genes that could be *targeted* by drugs!

What this means is that, looking at the genomes of hundreds of thousands of people, some of whom have some disease, such as heart disease, for example, I can find those mutations that are often seen in people with heart disease, but not seen very often in healthy people. This hints at there being something (such as a gene) near that mutation that influences heart disease in some way.

I can only do so much with the help of data, but once I have identified potential drug target genes, people working in the pharma industry can take this suggestion forward and start doing experiments in the lab to figure out if what the data told me stands up in the face of experiments – and if it does, they can take it further and try to develop drugs that target that gene.

As you can probably tell, this process takes a very long time, and often leads to dead ends – but without any clues from genetics, it used to take even longer, and it used to cost even more money, simply because we did not even know where to start then!