Populærvitenskapelig sammendrag
Primary adrenal insufficiency (PAI), or autoimmune Addison's disease (AAD), results from an impairment of the adrenal cortex to maintain a sufficient production of corticosteroid hormones. Patients with AAD depend upon a lifelong replacement therapy regime that is far from optimal, as no synthetic steroid suitably mimic the diurnal and ultradian pattern of cortisol. Therefore, a demand for better treatment solutions clearly exists, which requires a deeper understanding of the disease causing mechanisms. In this context, this project aims to generate steroid-producing cells by reprogramming urine-derived stem cells from patients, in order to observe the pathogenesis of AAD, in vitro. This is highly significant as adrenal tissue biopsy material from these patients is virtually impossible to obtain. Therefore, this induced steroidogenic cell line provides an otherwise unavailable source of cells that can recapitulate the genetics and the pathobiology of AAD patients.
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