Entries by The Snow Foundation

Dr. Vania Broccoli San Raffaele Hospital/CNR-Institute of Neuroscience in Milan, Italy. Eye gene therapy for restoring WFS1 gene function in Wolfram mice

Dr. Vania Broccoli San Raffaele Hospital/CNR-Institute of Neuroscience in Milan, Italy. Eye gene therapy for restoring WFS1 gene function in Wolfram mice AAV-based gene therapy is based on intra-vitreal injections of AAV vector expressing a therapeutic gene. This strategy is already in clinical use for treating Leber hereditary optic neuropathy (LHON) and clinical trials performed […]

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Dr. Rosemary Cater

Understanding the molecular role of WFS1 and how mutations in this protein cause Wolfram syndrome. Wolfram syndrome manifests as a genetic disorder marked by early-onset diabetes, progressive optic atrophy, and hearing loss. In addition to these defining symptoms, some individuals may also experience neurological complications, including motor impairments, neurological disorders, and deficits in memory and […]

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