DELPRAT Benjamin
Project status: active
Wolfram syndrome is a rare neurodegenerative disease leading to optic atrophy, hearing loss and diabetes. Other symptoms may also be present. This form of hereditary optic atrophy is very severe, as patients die at around the age of 35 of respiratory distress or pulmonary aspiration. Unfortunately, no medication currently exists.
The need to find a treatment is therefore urgent. Since Wolfram syndrome is a pathology affecting several organs, small active molecules could be used to effectively treat all the symptoms. Interestingly, zebrafish have become powerful tools for discovering new effective drugs for humans.
The objective of Dr. Delprat's project is to use a mutant zebrafish model for the gene responsible for Wolfram syndrome. This model, which develops visual and auditory deficits similar to those observed in patients, will make it possible to test a panel of effective molecules capable of slowing or stopping the disease, and open up prospects for a rapid clinical transfer to humans.
Doctor Benjamin Delprat
“Molecular Mechanisms in Neurodegenerative Dementia,” Montpellier University, France
Related scientific publication(s):
- Lucie Crouzier, Elodie M Richard, Jo Sourbron, Lieven Lagae, Tangui Maurice, Benjamin Delprat. Use of Zebrafish Models to Boost Research in Rare Genetic Diseases. Int J Mol Sci. 2021 Dec 12;22(24):13356. doi: 10.3390/ijms222413356.
- Lucie Crouzier, Alberto Danese, Yuko Yasui, Elodie M Richard, Jean-Charles Liévens, Simone Patergnani, Simon Couly, Camille Diez, Morgane Denus, Nicolas Cubedo, Mireille Rossel, Marc Thiry, Tsung-Ping Su, Paolo Pinton, Tangui Maurice, Benjamin Delprat. Activation of the sigma-1 receptor chaperone alleviates symptoms of Wolfram syndrome in preclinical models. Sci Transl Med. 2022 Feb 9;14(631):eabh3763.doi: 10.1126/scitranslmed.abh3763. Epub 2022 Feb 9.
- Lucie Crouzier, Elodie M Richard, Camille Diez, Hala Alzaeem, Morgane Denus, Nicolas Cubedo, Thomas Delaunay, Emily Glendenning, Sarah Baxendale, Jean-Charles Liévens, Tanya T Whitfield, Tangui Maurice, Benjamin Delprat. Morphological, behavioral and cellular analyses revealed different phenotypes in Wolfram syndrome wfs1a and wfs1b zebrafish mutant lines. Hum Mol Genet. 2022 Mar 21;ddac065. doi: 10.1093/hmg/ddac065. Online ahead of print.