Discovery of a drug candidate for GLIS3-associated diabetes

GLIS3 mutations are associated with type 1, type 2, and neonatal diabetes. Here, the authors generate mono-hormonal glucose-responding pancreatic β-like cells in vitro and through a screen identify a drug that rescues pancreatic β-like cell death in GLIS3 mutants by inhibiting the abnormally activat...

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Autores principales: Sadaf Amin, Brandoch Cook, Ting Zhou, Zaniar Ghazizadeh, Raphael Lis, Tuo Zhang, Mona Khalaj, Miguel Crespo, Manuradhi Perera, Jenny Zhaoying Xiang, Zengrong Zhu, Mark Tomishima, Chengyang Liu, Ali Naji, Todd Evans, Danwei Huangfu, Shuibing Chen
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2018
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Acceso en línea:https://doaj.org/article/c14e49f0fd174b1ebce0f8854fb56b94
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Sumario:GLIS3 mutations are associated with type 1, type 2, and neonatal diabetes. Here, the authors generate mono-hormonal glucose-responding pancreatic β-like cells in vitro and through a screen identify a drug that rescues pancreatic β-like cell death in GLIS3 mutants by inhibiting the abnormally activated TGFβ pathway.