Article

WFS1 gene delivery rescues visual function in a mouse model of Wolfram syndrome

2025-10-05

Abstract excerpt

<title>Abstract</title> <p> Wolfram syndrome is a rare childhood neurodegenerative disease characterized by diabetes followed by severe and rapid optic atrophy leading to blindness before the age of 20. Patients often develop other symptoms, such as deafness and neurological dysfunction. Wolfram syndrome is caused by mutations in the <italic>WFS1</italic> gene, which encodes wolframin protein. Despite decades...

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Literature Corpus work: 1587ff31-7aa0-50a3-9f2c-fb620758638f
DOI: 10.21203/rs.3.rs-7424550/v1

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