Focus

MOLECULAR BIOLOGY: TREATMENT DESIGN FOR DUCHENNE MUSCULAR DISTROPHY

Duchenne muscular dystrophy (DMD) is a severe condition characterised by muscle loss and causd by the absence of the cytoskeletal protein dystrophin. The utrophin protein can in part replace distrophyn and alleviate the symptoms associated with dystrophin loss in DMD patients. In this work the IBPM team led by Claudio Passananti sought to obtain therapeutic levels of utrophin expression in dystrophic muscles. They developed artificial transcriptiopn factors based on a particular module, called zinc finger (ZF ATFs) to activate utrophin expression. Passananti and colleagues engineered a particular ZF ATF called 'Jazz': they show that Jazz restores the integrity of the muscle sarcolemma and prevents the development of the dystrophic disease in dystrophin-deficient (mdx ) mice. These findings indicate that utrophin-based therapy can be efficiently developed using ZF ATF technology to treat DMD.