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Zebrafish Screen of compounds for individual or combination treatment of WS

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.
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Dr. Benjamin Delprat | University of Montpellier, France

Dr. Benjamin Delprat

University of Montpellier, France

 

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.

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