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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Detalles Bibliográficos
Autores principales: Kitamura, Rie Asada, Maxwell, Kristina G., Ye, Wenjuan, Kries, Kelly, Brown, Cris M., Augsornworawat, Punn, Hirsch, Yoel, Johansson, Martin M., Weiden, Tzvi, Ekstein, Joseph, Cohen, Joshua, Klee, Justin, Leslie, Kent, Simeonov, Anton, Henderson, Mark J., Millman, Jeffrey R., Urano, Fumihiko
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9675478/
https://www.ncbi.nlm.nih.gov/pubmed/36134655
http://dx.doi.org/10.1172/jci.insight.156549

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9675478/
https://www.ncbi.nlm.nih.gov/pubmed/36134655
http://dx.doi.org/10.1172/jci.insight.156549

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