Gene therapy reverses heart failure in mouse model of Barth syndrome

Barth syndrome is a rare genetic disease in boys that can cause life-threatening heart failure and also weakens the skeletal muscles and the immune system. There is no specific treatment, but new research, involving new mouse models, shows the potential of a gene therapy approach in preventing and reversing cardiac dysfunction in Barth syndrome.

Source: Gene therapy reverses heart failure in mouse model of Barth syndrome