Two ‘remarkable’ new gene therapy studies have shown ‘tremendous’ progress in treating deafness and blindness, according to Professor Luke O’Neill.

On The Pat Kenny Show this morning, the Trinity professor said the two studies could eventually benefit 'millions and millions' of people around the world.

The first study, carried out at Fudan University in Shanghai and published in Nature Medicine, saw five children who were born deaf hearing in both ears for the first time.

The children were unable to hear because of inherited genetic mutations that left them unable to create a protein needed to properly transmit auditory signals from the ear to the brain.

The therapy uses an inactive virus to smuggle working copies of the affected gene, prompting cells in the ear to generate working copies of the protein.

Within weeks the children had gained hearing and could locate the sources of sounds.

Two of them were even recorded dancing to music.

'Tremendous'

“Can you imagine?” asked Prof O’Neill. “They were deaf and suddenly they could hear again.”

“The trick was both ears were done. It was the first time ever that both the left and right ear were done.”

He warned that the therapy remains experimental and risks remain, but added: “So far, this shows safety is really good in this as well, which is tremendous.”

“It's a big one – there are 430 million people in the world who have some kind of deafness, 26 million from birth and 60% is genetic.

“That's the sort of numbers we're talking about. So ultimately. This could benefit millions and millions of children.”

Vision

The second study, led by Harvard Medical School researchers and published in the New England Journal of Medicine.

The trial used CRISPR gene editing in a bid to improve vision in people with inherited blindness – and recorded measurable improvement in 11 of the 14 people taking part.

The study's main focus was to test whether the approach was safe and effective, with researchers finding the approach had encouraging results and no harmful side effects.

Prof O’Neill said one of the women involved in the trial can now see things she had never seen before.

“First of all, they selected these people out with this inherited retinal degeneration and they gave them the injection,” he said.

“Then again, over the course of a few weeks, they began to test their vision and what I love, this one woman said - she was blind, remember - and three months later, she could see Christmas tree lights twinkling.

“Now, can you imagine you've been blind for years and suddenly she could see these Christmas tree lights twinkling.”

He noted that the treatment does not fully restore vision – but some of the people involved can now make out faces or see food on a plate.

“Can you imagine? Isn't that tremendous?” he said.

“In other words, it's a big step down the road towards restoring vision.”

CRISPR

Prof O’Neill noted that CRISPR has still only been tested on around 250 people around the world and the technique remains highly experimental.

He said both studies are “causing huge excitement” in the drug industry.

“You're restoring hearing and vision in children, which is a tremendous goal,” he said.

“I have no doubt that this will galvanise, increasingly now, companies to get more involved.”