New breakthroughs in gene editing mean that cures for genetic diseases such as cystic fibrosis could be developed in the near future.

Gene editing technology has advanced significantly recently, off the back of a 2012 discovery.

On this week’s Show Me The Science podcast, Professor Luke O’Neill said that the ability to edit genes began with the discovery of CRISPR technology.

“CRISPR was discovered by two scientists, Jennifer Doudna and Emmanuelle Charpentier... They won the Nobel Prize for this discovery, it was discovered in 2012,” he said.

“The CRISPR mechanism was first seen in bacteria – often, scientists use different organisms to study things.

“What these two people discovered was the bacteria make an enzyme that can attack viral genes.

“Now, virus infect bacteria – virus affect all of us, of course.

“Our while life on Earth is affected by viruses and have genes – and it turns out bacteria have a defence mechanism against viruses.

“So, the virus goes into the bacteria - the bacteria makes this thing called CAS - CAS locks onto the viral DNA and edits it, chops it out basically.”

Prof O’Neill said that this discovery can be used to clip and correct genes in various ways.

Micropipette and Eppendorf microcentrifuge tubes, DNA sequencing in background. Image: Connect Images / Alamy. 15 January 2014

According to Prof O’Neill, the first approval for a CRISPR gene editing therapy was handed out in the UK in November for the treatment of sickle cell anaemia, which limits the body’s supply of haemoglobin.

“People with this disease, it’s awful, very painful,” he said.

“Then, the severe end, you can die from it, right? So, the question became, could you correct that broken gene?

“The gene is broken – you're getting broken haemoglobin and now these sickle cells are there and they can’t carry oxygen and you get sickle cell disease.

“There’s a second disease called transfusion dependent beta thalassemia – and again, that’s a dodginess in the gene for the haemoglobin as part of this as well.

“The CRISPR people thought, can we correct the haemoglobin? And indeed we can.

“What they do here is they correct the broken gene and now you get a normal haemoglobin and then you’re fine and you don’t have sickle cell disease anymore.”

However, Prof O’Neill said that with this method, there had been a concern that the wrong gene could be hit.

New breakthrough

Now though, Lithuanian scientists have come up with a new technology which massages the gene instead of chopping it out.

“It relaxes the DNA double helix and now the gene doesn’t work, doesn’t get expressed,” Prof O’Neill said.

“If you take it away, the gene goes back to normal. So what we have here is a reversible way to do this.

“Just in case something untoward happens, you can withdraw the therapy, and so therefore it’s likely to be a bit safer – this is the idea.”

According to Prof O’Neill, this new development could help to treat a range of genetic diseases in the near future.

Featured image: Split image showing Luke O'Neill (L) and red DNA strands (R).