National Research Council of Italy: Project application
Title: Novel experimental therapies to treat blindness in Wolfram syndrome
Applicant: Dr. Vania Broccoli
San Raffaele Hospital/CNR-Institute of Neuroscience, Milan Italy
Eye gene therapy for restoring WFS1 gene function in Wolfram mice
Extending into 2026:
- This work will be done in conjunction with a grant from the Be a Tiger Foundation
- This 12-month project aims to determine which cells in the retina will need to be the target of gene editing or gene therapy vectors.
- Understanding which retinal cells are affected will help gene editing provide a more complete cure for visionloss in Wolfram syndrome.