Multidimensional analysis and therapeutic development using patient iPSC–derived disease models of Wolfram syndrome

Multidimensional analysis and therapeutic development using patient iPSC–derived disease models of Wolfram syndrome

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 syndrome as a spectrum disorder. Therefore, a gen...

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Bibliographic Details
Main Authors: Rie Asada Kitamura, Kristina G. Maxwell, Wenjuan Ye, Kelly Kries, Cris M. Brown, Punn Augsornworawat, Yoel Hirsch, Martin M. Johansson, Tzvi Weiden, Joseph Ekstein, Joshua Cohen, Justin Klee, Kent Leslie, Anton Simeonov, Mark J. Henderson, Jeffrey R. Millman, Fumihiko Urano
Format: Article
Language:English
Published: American Society for Clinical investigation 2022-09-01
Series:JCI Insight
Subjects:
Endocrinology
Genetics
Online Access:https://doi.org/10.1172/jci.insight.156549

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