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WFS1 Gene Therapy to correct chronic inflammation due to Wolframin loss of function mutations

Principal Investigators- Drs. Raniero Chimienti and Giulio Frontino, Milan, Italy

  • This work is done in collaboration with Telethon Foundation, Italy
  • This project is designed to test whether immune cells that have been genetically corrected can help improve the inflammatory symptoms of Wolfram syndrome in a mouse model.
  • By transplanting genetically corrected WS immune cells into mice with Wolfram syndrome, researchers will study whether inflammation is reduced and whether the progression of Wolfram syndrome symptoms can be slowed by addressing/reducing inflammation.
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Dr. Maurizio Delvecchio and Dr. Giulio Frontino’s Update | San Raffaele Hospital, Milan, Italy

Dr. Giulio Frontino and Dr. Maurizio Delvecchio discuss crafting national guidelines for the diagnosis and care of individuals with Williams Syndrome. You can listen to their presentation by following the link provided: https://youtu.be/PssLjWZgsFE

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