TY - T1 - Stem Cell Based Models in Congenital Hyperinsulinism - Perspective on Practicalities and Possibilities SN - / UR - http://hdl.handle.net/10138/342375 T3 - A1 - Lithovius, Väinö; Otonkoski, Timo A2 - PB - Y1 - 2022 LA - eng AB - Congenital hyperinsulinism (CHI) is a severe inherited neonatal disorder characterized by inappropriate insulin secretion caused by genetic defects of the pancreatic beta cells. Several open questions remain in CHI research, such as the optimal treatment for the most common type of CHI, caused by mutations in the genes encoding ATP-sensitive potassium channels, and the molecular mechanisms of newly identified CHI genes. Answering these questions requires robust preclinical models, particularly s... VO - IS - SP - OP - KW - congenital hyperinsulinism; stem cell derived islets; disease modeling; drug screening and discovery; insulin secretion; hypoglycemia; genetic defects; PANCREATIC BETA-CELLS; INSULIN-SECRETION; IN-VITRO; HYPOGLYCEMIA; MUTATION; MICE; GENERATION; INDUCTION; ISLETS; PROLIFERATION; 3121 General medicine, internal medicine and other clinical medicine N1 - PP - ER -