Nature Communications (Jul 2018)

Discovery of a drug candidate for GLIS3-associated diabetes

  • 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

DOI
https://doi.org/10.1038/s41467-018-04918-x
Journal volume & issue
Vol. 9, no. 1
pp. 1 – 12

Abstract

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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.