Multidimensional analysis and therapeutic development using patient iPSC–derived disease models of Wolfram syndrome
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DOI: 10.1172/jci.insight.156549
Abstract: Wolfram syndrome is a rare genetic disorder largely caused by pathogenic variants in the WFS1 gene and manifested by diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration. Recent genetic and clinical findings have revealed Wolfram… read more here.
Keywords: ipsc derived; therapeutic development; analysis; wfs1 1672c ... See more keywords