It is possible that other mutations may be found in the future with similar or even superior beneficial potential to the mutated gene currently under investigation. Madeddu and Puca believe their study could lead to a new wave of treatments inspired by the genetics of centenarians.
“Our findings confirm that the healthy mutated gene can reverse the decline in heart function in older people. We now want to find out whether administering the protein instead of the gene also works. Gene therapy is widely used to treat diseases caused by bad genes, but a protein-based treatment is safer and easier to perform than gene therapy,” said Madeddu. The protein that would then be administered is the protein encoded by the mutated gene.
“We have received funding from the Medical Research Council to test healthy gene therapy for progeria. Also known as Hutchinson-Guilford syndrome, this genetic disease causes early age damage to the heart and blood vessels of children. We have also received funding from the British Heart Foundation and Diabetes UK to test the protein in elderly and diabetic mice,” he said.
Gene therapy with the healthy gene in mouse models has already been shown to prevent the onset of atherosclerosis [verharding van de slagaders door plaques]prevents aging of the blood vessels and complications of diabetes and rejuvenates the immune system,” said Puca. “We have new confirmation and enhancement of the gene/protein’s therapeutic potential.”
The researchers hope to be able to start clinical studies on patients with heart failure soon.
The researchers’ study is published in Cardiovascular Research. This article is based on a press release from the University of Bristol and a telex from the IPS news agency.