Article
Comprehensive overview of disease models for Wolfram syndrome: toward effective treatments.
Mammalian genome : official journal of the International Mammalian Genome Society - 1 Mar 2024
Morikawa Shuntaro, Tanabe Katsuya, Kaneko Naoya, Hishimura Nozomi, Nakamura Akie
Abstract excerpt
Wolfram syndrome (OMIM 222300) is a rare autosomal recessive disease with a devastating array of symptoms, including diabetes mellitus, optic nerve atrophy, diabetes insipidus, hearing loss, and neurological dysfunction. The discovery of the causative gene, WFS1, has propelled research on this disease. However, a comprehensive understanding of the function of WFS1 remains unknown, making the development of...
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