ESPE Abstracts (2019) 92 S7.1

Novel Interventions to Treat Adrenal Insufficiency

Leonardo Guasti

Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, United Kingdom

The generation of functional, long-lived adrenocortical cells through reprogramming from pluripotent stem cells or via lineage conversion strategies, could offer an alternative treatment modality for adrenal insufficiency (AI) due to a variety of causes, such as mutation in steroidogenic enzymes as seen in congenital adrenal hyperplasia (CAH). The recent generation of human induced steroidogenic cells (hiSCs) from fibroblasts, blood- and urine-derived cells established from healthy donors and patients affected by CAH has provided a new paradigm for disease modelling and potentially cell therapy. For the latter, further research is needed using appropriate AI models. We will be discussing the design of reprogramming protocols compatible with clinical applications, the effort in establishing patient-derived adrenocortical organoids (which adrenocortical stem cell antigen can be used to generate organoids from a single cell?) and the application of gene editing technologies on adrenocortical cells in vivo or ex vivo.

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