Persistent hyperinsulinaemic hyperglycaemia of infancy-derived cells; implications for β-cells that replicate in vitro

Abstract

Our recent work upon a rare disease has established proof of the concept that in vitro gene therapy could be used to successfully reverse a metabolically related disorder. Our results allude to the possibility that in future, following pancreatectomy, acutely isolated β-cells from PHHI patients could be similarly engineered for subsequent autotransplantation. By transgenic manipulation of the NES2Y cells, we have generated the first fully glucose-responsive human insulin-secreting cell line. We believe that these, and other PHHI-derived islet cell lines, will be of major importance for in vitro studies of human β-cell function and potentially valuable in transplantation-based therapies for both diabetes mellitus and PHHI.