Article Correctness Is Author's Responsibility: Gene therapy reverses heart failure in mouse model of Barth syndrome

(Boston Children's Hospital) 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 at Boston Children's Hospital, involving new mouse models, shows the potential of a gene therapy approach in preventing and reversing cardiac dysfunction in Barth syndrome.