The cardiomyopathy associated with muscular dystrophies is characterized clinically by effects on the pumping function of the heart, in which the principal complication is the occurrence of cardiac insufficiency and/or electrical abnormalities with problems of conduction or rhythm. These complications, along with respiratory complications, are the principal causes of death in these diseases.
Program for the treatment of cardiomyopathies using gene therapy
- Reference laboratory: Genethon
Gene therapy could provide important therapeutic tools for the treatment of these cardiac effects. Genethon has developed a program which aims to treat these forms of cardiomyopathies by administration of an AAV (Adeno Associated Virus) carrying a therapeutic gene.