Advances in gene editing “opens the door” to curing many more fatal diseases, Luke O’Neill. 

The Trinity Professor said scientists have rewritten a baby's DNA in order to cure it of carbamoyl phosphate synthetase 1 (CPS1) deficiency. 

On The Pat Kenny Show, Professor O’Neill said the success of the procedure is hugely consequential. 

“A baby with a very severe rare disease has now been effectively cured with gene editing,” he said. 

“It’s remarkable because it’s called CPS1 deficiency, there’s a single change in your DNA - they corrected that. 

“Normally, that disease would kill that baby within about a week - half the babies die within a week; nine and a half months later, this baby is pretty normal. 

“It’s seen as a real advance in terms of gene editing.” 

CPS1 only impacts one gene and is extremely rare, with only about one in every 1.3 million babies born with the condition. 

“A single gene is broken with this disease and what that means is your body can’t handle ammonia,” Professor O’Neill said. 

“We make ammonia in our own bodies and that builds up and becomes toxic, it causes brain damage and liver damage and so on.” 

Other diseases

While CPS1 changes only one gene out of the tens of thousands within a human’s body, the condition has a severe impact on a baby’s health. 

“Within about a month, there’s a lack of development in the brain, the liver doesn’t develop properly and it’s really fatal,” Professor O’Neill said. 

“There is a drug that can be used to treat it which can limit it a bit but this seems to be the way to do it.

“The reason it’s so exciting is it opens the door to many diseases where your genes are different.” 

Main image: Luke O'Neill. Picture by: Newstalk.