Article

Liraglutide treatment reverses unconventional cellular defects in induced pluripotent stem cell-derived β cells harboring a partially functional WFS1 variant

2024-08-07

Abstract excerpt

<h4>Aims/hypothesis</h4> Wolfram Syndrome 1 (WS1) is a rare genetic disorder characterized by very heterogeneous clinical manifestations caused by variants of the WFS1 gene, which encodes for the Endoplasmic Reticulum (ER) protein Wolframin, involved in cellular stress response, Ca 2+ handling and autophagy. Given the central role of Wolframin, elucidating the impact of WFS1 variants on cell functions is cruci...

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Literature Corpus work: 4941f97c-5ab7-5024-8643-94e6afbb08f3
DOI: 10.1101/2024.08.06.606801

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