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Congenital hyperinsulinism caused by mutations in the K ATP -channel-encoding genes (K ATP HI) is a potentially life-threatening disorder of the pancreatic beta cells. No optimal medical treatment is available for patients with diazoxide-unresponsive diffuse K ATP HI. Therefore, we aimed to create a model of K ATP HI using patient induced pluripotent stem cell (iPSC)-derived islets.

SUR1-mutant iPS cell-derived islets recapitulate the pathophysiology of congenital hyperinsulinism