GLP-1R agonists demonstrate potential to treat Wolfram syndrome in human preclinical models.
Abstract | AIMS/HYPOTHESIS: METHODS: The effect of the GLP-1R agonists dulaglutide and exenatide was examined in Wfs1 knockout mice and in an array of human preclinical models of Wolfram syndrome, including WFS1-deficient human beta cells, human induced pluripotent stem cell (iPSC)-derived beta-like cells and neurons from control individuals and individuals affected by Wolfram syndrome, and humanised mice. RESULTS: CONCLUSIONS/INTERPRETATION: Our study provides novel evidence for the beneficial effect of GLP-1R agonists on WFS1-deficient human pancreatic beta cells and neurons, suggesting that these drugs may be considered as a treatment for individuals with Wolfram syndrome.
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Authors | Vyron Gorgogietas, Bahareh Rajaei, Chae Heeyoung, Bruno J Santacreu, Sandra Marín-Cañas, Paraskevi Salpea, Toshiaki Sawatani, Anyishai Musuaya, María N Arroyo, Cristina Moreno-Castro, Khadija Benabdallah, Celine Demarez, Sanna Toivonen, Cristina Cosentino, Nathalie Pachera, Maria Lytrivi, Ying Cai, Lode Carnel, Cris Brown, Fumihiko Urano, Piero Marchetti, Patrick Gilon, Decio L Eizirik, Miriam Cnop, Mariana Igoillo-Esteve |
Journal | Diabetologia
(Diabetologia)
Vol. 66
Issue 7
Pg. 1306-1321
(07 2023)
ISSN: 1432-0428 [Electronic] Germany |
PMID | 36995380
(Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
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Copyright | © 2023. The Author(s). |
Chemical References |
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Topics |
- Humans
- Animals
- Mice
- Wolfram Syndrome
(drug therapy, genetics)
- Exenatide
(therapeutic use)
- Induced Pluripotent Stem Cells
- Optic Atrophy
(pathology)
- Insulin-Secreting Cells
(pathology)
- Mice, Knockout
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