Gene therapy has slowed life-threatening heart disease

In a new study, mice with lost PKP2 function were shown to survive for more than five months if they received a single dose of gene therapy carrying a normal version of the gene. Rodents that received the replacement gene also showed a 70 to 80 per cent reduction in fibrotic tissue formation, depending on the dose.

The therapy can significantly slow the disease and prevent tissue loss, since in advanced stages the heart is already so affected that a transplant is often necessary.