Principal Investigator Dr.Benjamin Delprat, Montpellier, France
With help from the WS patient community and a special shout-out to our dear friend Nufar, a list of 30 widely available and potentially therapeutic compounds was compiled to be evaluated for the potential treatment of WS.
We are completing negotiations to test these compounds individually and in combination in a WS zebrafish model.
We aim to determine the most effective and accessible compounds that may serve as a treatment “cocktail” to aid in slowing the progression of symptoms in WS.
http://thesnowfoundation.org/wp-content/uploads/2019/06/snow-foundation_logo.svg00The Snow Foundationhttp://thesnowfoundation.org/wp-content/uploads/2019/06/snow-foundation_logo.svgThe Snow Foundation2026-02-19 20:06:572026-02-19 20:06:57Zebrafish Screen of compounds for individual or combination treatment of WS
In my lab, we are developing concomitantly two therapeutic strategies: a pharmacological approach and a gene therapy. To achieve these goals, we are working with suited animal models: two transgenic mouse lines and one zebrafish line. One mouse model and the zebrafish line are deficient for Wolframin, the protein responsible for Wolfram syndrome type 1. The other mouse model has been genetically engineered to mimic a human mutation, recapitulating sensorial deficits (vision and hearing loss) and diabetes. We are hoping to treat vision and hearing, as well as central neurodegeneration.
http://thesnowfoundation.org/wp-content/uploads/2019/06/snow-foundation_logo.svg00The Snow Foundationhttp://thesnowfoundation.org/wp-content/uploads/2019/06/snow-foundation_logo.svgThe Snow Foundation2024-03-29 19:46:232024-10-12 09:53:46Dr. Benjamin Delprat | University of Montpellier, France
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