Abstract
Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.
Highlights
Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms
Dr Hattersley’s team and ours identified several dominant de novo Wolfram syndrome 1 (WFS1) variants associated with a genetic syndrome of neonatal/infancy-onset diabetes, congenital sensorineural deafness, and congenital cataracts [9]
WFS1 is a locus of broad interest to various disease processes, highlighting the need for therapeutics targeting the gene towards potential treatments for Wolfram-syndrome-spectrum disorders
Summary
Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. We summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder. Dr Hattersley’s team and ours identified several dominant de novo WFS1 variants associated with a genetic syndrome of neonatal/infancy-onset diabetes, congenital sensorineural deafness, and congenital cataracts [9]. WFS1 is a locus of broad interest to various disease processes, highlighting the need for therapeutics targeting the gene towards potential treatments for Wolfram-syndrome-spectrum disorders
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