Genome editing: Cut and paste

Every year the Society gives out a number of prizes to leading geneticists, who win the opportunity to give a lecture about their work. Andrew Wood, from the University of Edinburgh, is the winner of this year’s Balfour lecture, given to an outstanding young investigator. Here he is talking to Ginny Smith about his prize-winning work...

Andrew - CRISPR-Cas9 is a new tool. It was developed for use in human cells in 2012. However, it replaced older, clunkier versions of technology that aim to do essentially the same thing. and part of what I was talking about today was my experience with some of those older technologies and comparisons between those and modern-day methods.

Ginny - So what kinds of sequences might you be wanting to add to or take out of the genome and why?

Andrew - Well for example, let’s imagine that a clinician has identified a human disease and a gene variant has been found in individuals who have that disease. In order to show that that gene variant is causal for the disease, one of the approaches that researchers take is to take cells and engineer that particular variant into a cell that didn’t have it previously and to see whether or not that cell behaves differently in a way that might link that variant to the disease identified in the human.

Ginny - So you can actually kind of create models of these diseases in animals?

Andrew - In animals, in human cells, essentially, any living organism now. That’s one of the great things about genome editing, that it is portable between just about any living system that you can imagine.

Ginny - And I guess that then means that you can test drugs and other therapies, and see if you can cure the disease that you’ve created?

Andrew - Absolutely. It means that you can create for example, cellular models that have these variants and then screen compounds or drugs and see how cells that have that variant behave and how that differs from cells that don’t have that variant. That would be one approach that a lot of people are taking.

Ginny - Is there anything in particular that you’ve come across so far in this meeting or that’s coming up tomorrow that you think is really exciting and you think might be the kind of the next step in genetics and healthcare?

Andrew - The general theme of mining large quantities of DNA sequence data in order to find out what distinguishes populations of individuals with respect to their susceptibility to diseases I think is really exciting.

Ginny - So looking at the kind of preventative or predicting disease before it even happens rather than curing it after it’s happened?

Andrew - Yeah, exactly that. Also, perhaps kind of retrospectively looking at the way that different groups of patients have responded to a particular drug and seeing whether or not those which responded in a positive manner have particular variants that are not present in the group that didn’t respond well to the drug for example.

Ginny - So this is coming to us to a kind of idea of personalised healthcare that we’ll all be screened and whatever treatments we’re given will be tailored for us.

Andrew - Absolutely, yeah. That’s a big buzz word in medicine at the moment.

Ginny - Do you think it’s likely that that’s going to be happening soon or is it still a long, long way away in terms of being financially viable?

Andrew - Well, I mean there are a few sort of poster child cases that get kind of trotted out as a good test case for this kind of approach. It is inherently a very expensive business. First of all gathering the genetic information is very expensive. Any kind of drug development process is also incredibly expensive. So yes, the idea of this leading to therapies quickly that can be applied to lots of people at the moment still seems quite a long way away.