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CELL-MYOME

Over the past few years the development of therapies for muscular dystrophies has reached a new momentum and a number of novel strategies have been developed to correct the deleterious effects of mutations. The aim of the project is to determine and understand the molecular consequences of novel therapeutics on human disease cell models of muscular dystrophy such as Duchenne Muscular Dystrophy and Myotonic Dystrophy. A basic rationale is that prediction of the human clinical response demands testing in human cell models, to complement studies in animal models. To decipher the molecular inference of novel therapeutics on human, we propose to treat human disease muscle cell models and analyse their effects on gene expression by deep RNA-sequencing to determine entire transcriptomic modulations including alternative splicing events. Gene networking/interactome analyses will provide insight into the potential clinical application to human subjects.