Role of the Epigenome and noncoding Genome in Duchenne Muscle Dystrophy

Discovery of an "epigenetic" level of gene regulation, beyond DNA sequence, suggested that aside given genetic defects being associated with a specific pathology, global mechanisms involving various levels of chromosome structure and organization play a crucial role in determining the real complexity of why cells lose their identity and functions. The goal of this research program is to fill the gap between the defined genetic level that characterize Duchenne Muscle Dystrophy and epigenetic mechanisms that cooperate to the onset and progression of this disease. Noncoding portions (repetitive and epigenetic elements) and (RNA) that characterizes most of the structure and output of the human genome is thought to play a fundamental role in epigenome regulation. Hence, in depth investigation of this emerging and largely mysterious world will prove to be mandatory for the comprehension, possible early diagnosis and potential treatment to ameliorate prognosis of this as well as other degenerative diseases.