Development of novel human stem cell models of Beta-Propeller Protein- Associated Neurodegeneration (BPAN) for disease modelling and drug screening

Awardee: Paul Lockhart

Institution: Murdoch Children's Research Institute

Award Amount: $61,245

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Can we improve neurologic and psychiatric outcomes in MSUD? Studies in a new mouse model

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Pre-clinical animal model and cell evaluation of candidate therapies for human NUBPL disease.