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Mariana Igoillo-Esteve – Centre pour la Recherche du Diabète, ULB: GLP-1 analogs for the treatment of Wolfram syndrome.


2016 - Wolfram syndrome is a rare genetic disorder, also known as DIDMOAD syndrome. It is caused by mutations in the WFS1 gene, coding for a protein called Wolframin localized in the endoplasmic reticulum. The main clinical manifestations of the disease appear in childhood and are diabetes mellitus, diabetes insipidus, optic nerve atrophy and deafness. In early adulthood, the patients also develop neurological disorders including ataxia, breeding problems and depression. The prognosis of the disease is poor, and there are currently no effective treatments. The molecular pathogenesis of Wolfram syndrome has been investigated using in vitro (cellular) and in vivo (mouse) models. These studies have shown that wolframin deficiency results in endoplasmic reticulum stress in pancreatic beta cells and neurons, leading to cell dysfunction and death. GLP-1 analogs are drugs currently used for the treatment of type 2 diabetes. We hypothesize that these molecules can increase the cellular defenses against endoplasmic reticulum stress in Wolfram syndrome, and prevent or delay pancreatic beta cell and retinal neuro-degeneration. To test this hypothesis, we will make use of disease-relevant in vitro and in vivo experimental approaches. Based on the outcomes of the present preclinical project, studies assessing GLP-1 analogs treatment in Wolfram syndrome patients may be considered.

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