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Gene Therapy Treatment of Wolfram Syndrome

Principal investigator Dr. Cécile Delettre-Cribaillet, INM, Montpelier, France

Extending into 2026:

  • This work will be done in conjunction with a grant from the Be a Tiger Foundation
  • This is an ongoing project evaluating and validating the potential for WFS1 wildtype gene transfer as atreatment for Wolfram syndrome.
  • Preclinical data have already shown the ability of gene therapy to correct symptoms of Wolfram syndrome in a WFS1 knock-out model.
  • New steps in the project include ensuring that overexpression of WFS1 in cells will not impair this correction and validating the already reported promising effects of gene therapy in a knock-in mouse model that more precisely mirrors human WS.
  • This complete data set will help us to determine the efficacy of gene therapy for preserving vision in WS
  • This work will provide validation and efficacy data to move gene therapy closer to development for WS.
  • Corrected WFS1 will be delivered with a retina-specific vector, directly to the retina with direct injection to the eye in a WS knock-in (more similar to humans than a knock-out) mouse model.
  • Corrected WFS1 will be delivered systemically with a different vector, also in a knock-in mouse model, to determine if multiple organs (including pancreas and retina) can be treated in WS with a single treatment.
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Dr. Cecile Delettre | Neuroscience Institute of Montpellier, France

Dr. Cecile Delettre

Neuroscience Institute of Montpellier, France

Recently we have identified a family of molecules with the capacity to stimulate significantly the growth of retinal ganglion cells in vitro in a model of optic atrophy. We developed a zebrafish model with an optic atrophy and we have treated these fishes with one of these molecule. We used increasing doses of molecules to measure the toxicity and determine the most effective dose to protect the optic nerve. We have determined the dose with the best effect and confirmed that treatment with this molecule can prevent optic nerve developmental delay in vivo in our model of optic neuropathy. We are studding the mechanism of action of this molecule and using it in several model of Wolfram syndrome We look forward to a clear indication of the possibility of using this molecule in the future development of a treatment for Wolfram syndrome.

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