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Pathogenic pathway - PowerPoint PPT Presentation

DISEASE GENE IDENTIFIED FOR DISORDER WITH PRESYMPTOMATIC WINDOW

SCREEN FOR:. DOWNREGULATING SMALL MOLECULES *. DOWNREGULATING ASOS **. GAIN OF FUNCTION. PROTEIN DESTABILIZERS/FUNCTIONAL INHIBITORS**. PATHOGENIC PATHWAY INHIBITORS. LOSS OF FUNCTION. CONFIRM IN DISEASE RELATED CELLULAR AND ANIMAL MODELS. SPLICING MUTATION. SPLICE MODULATING ASOS **.

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Pathogenic pathway - PowerPoint PPT Presentation

DISEASE GENE IDENTIFIED FOR DISORDER WITH PRESYMPTOMATIC WINDOW