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Calpain inhibitor and ibudilast rescue β cell functions in a cellular model of Wolfram syndrome

Wolfram syndrome is a rare multisystem disease characterized by childhood-onset diabetes mellitus and progressive neurodegeneration. Most cases are attributed to pathogenic variants in a single gene, Wolfram syndrome 1 (WFS1). There currently is no disease-modifying treatment for Wolfram syndrome, a...

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Bibliografske podrobnosti
izdano v:Proc Natl Acad Sci U S A
Main Authors: Nguyen, Lien D., Fischer, Tom T., Abreu, Damien, Arroyo, Alfredo, Urano, Fumihiko, Ehrlich, Barbara E.
Format: Artigo
Jezik:Inglês
Izdano: National Academy of Sciences 2020
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC7382278/
https://ncbi.nlm.nih.gov/pubmed/32632005
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.2007136117
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