Health · Medical Research · 4 hrs ago
MCRI researchers report gene therapy that reversed heart disease in animal studies
Murdoch Children’s Research Institute researcher Enzo Porrello presented gene therapy research at a New York event hosted by Friends of MCRI.
The work targets severe forms of heart disease that can lead to heart failure in children.
Porrello said heart disease affects about one in 100 children, or roughly 40,000 children born in the United States each year.
For children with heart failure, a transplant is the only treatment, but it is a last resort and not a cure.
In animal studies, a single injection prevented and reversed certain heart disease, according to Porrello.
The results have not yet been tested in clinical trials; MCRI’s next goal is to move the therapy into trials.
MCRI researchers say a gene therapy reversed established heart disease in animal studies.
Professor Enzo Porrello said the therapy could also prevent the disease from developing.
The treatment is a single-shot injection aimed at certain severe forms of childhood heart disease.
MCRI’s next focus is moving the therapy from the lab into clinical trials.
Porrello discussed the research at a Friends of MCRI event in New York.
- Who
- MCRI researchers, including Professor Enzo Porrello, who leads its Heart Regeneration Group.
- What
- They reported that a gene therapy prevented and reversed heart disease in animal studies. They aim to move it into clinical trials.
- When
- Porrello spoke at the event on Thursday night. The article was published on October 17, 2026.
- Where
- Old Mates in Manhattan, New York City.
- Why
- The researchers are seeking better, targeted treatments for severe heart disease in children that can lead to heart failure.
This story does not have two clearly opposing sides.
Porrello said MCRI researchers had been working on this area of research.
Researchers demonstrated that the gene therapy could prevent and reverse heart disease in animals.
Porrello discussed the research at the Friends of MCRI New York event.
MCRI’s focus is moving the therapy out of the lab and into clinical trials.
- Research institute
- Murdoch Children’s Research Institute (MCRI)
- Research lead
- Professor Enzo Porrello
- Treatment
- Single-shot gene therapy
- Study result
- Prevented and reversed established heart disease in animal studies
- Event venue
- Old Mates, Manhattan, New York City
- Next step
- Clinical trials
What people are saying
We’ve shown in animal studies that not only can this gene therapy prevent the development of heart disease
It can actually completely reverse established heart disease… which is incredible. We’ve never seen anything like that in our sort of 15 to 20 years of doing this work.
And so we’re super excited now to take that gene therapy out of the lab and get it into clinical trials.