Focal Treatment for Genetic Disorders (A Fairy Tale or a True Story?)

Genetic is widely involved in pathophysiology of many diseases. However, certain gene abnormalities could cause cardiac disorders called monogenic disorders (1). Since the genetic information and hence abnormal gene is present in all cells, treatments are focused on symptoms. Nowadays, gene transfer is an uprising option (2). Although all cells carry a mutant gene, the major effect of the disease is localized to a group of cells in some patients. Brugada syndrome results from a sodium channel mutation that causes ST elevation in right precordial leads and may lead to sudden ventricular fibrillation, especially in young males (3). This main electrocardiographic disturbance originates from RV epicardium (4). Therefore, some interventionists try to treat the disease by ablation of this area (5). Although implanting a defibrillator is yet the standard recommendation, it does not cure the disease (6).