Dr. Singh will apply artificial intelligence to PWS datasets to glean new information about pathways disrupted in PWS and possible targets for therapy.
Dr. Nicholls has identified deficits in a set of proteins that facilitate the folding and maturation of other proteins, ER chaperone proteins. He believes deficits of these proteins in the pancreas is an important contributor to endocrine dysfunction in PWS. Here he will assess the ability of drugs that activate these chaperone proteins to rescue...
Dr. Mitchell and her team have been investigating how beloranib, a drug that effectively reduced hyperphagia and induced weight loss in individuals with PWS, worked. By defining downstream effectors of beloranib’s hyperphagia-reducing action, they hope to identify a safe and effective drug to treat hyperphagia in PWS. Dr. Theresa Strong, Director...
Dr. Bang and her team will apply a series of ‘assays’ (lab tests evaluating cell function) to PWS patient-specific stem cells (iPSC) that have been driven to become cortical neurons in a lab dish. Once validated, these assays can be used to discover novel therapeutic targets for PWS, screen for drugs that can correct impaired neuronal function,...
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