Inhibition of pancreatic EZH2 restores progenitor insulin in T1D donor.

Keith Al-Hasani; Ishant Khurana; Lina Mariana; Thomas Loudovaris; Scott Maxwell; K N Harikrishnan; Jun Okabe; Mark E Cooper; Assam El-Osta
Abstract
Type 1 diabetes (T1D) is an autoimmune disease that selectively destroys insulin-producing β-cells in the pancreas. An unmet need in diabetes management, current therapy is focussed on transplantation. While the reprogramming of progenitor cells into functional insulin-producing β-cells has also been proposed this remains controversial and poorly understood. The challenge is determining why default transcriptional suppression is refractory to exocrine reactivation. After the death of a 13-year-old girl with established insulin-dependent T1D, pancreatic cells were harvested in an effort to restore and understand exocrine competence. The pancreas showed classic silencing of β-cell progenitor genes with barely detectable insulin (Ins) transcript. GSK126, a highly selective inhibitor of EZH2 methyltransferase activity influenced H3K27me3 chromatin content and transcriptional control resulting in the expression of core β-cell markers and ductal progenitor genes. GSK126 also reinstated Ins gene expression despite absolute β-cell destruction. These studies show the refractory nature of chromatin characterises exocrine suppression influencing β-cell plasticity. Additional regeneration studies are warranted to determine if the approach of this n-of-1 study generalises to a broader T1D population.
Journal SIGNAL TRANSDUCTION AND TARGETED THERAPY
ISSN 2059-3635
Published 22 Jul 2022
Volume 7
Issue 1
Pages 248
DOI 10.1038/s41392-022-01034-7
Type Case Reports | Journal Article | Research Support, Non-U.S. Gov't
Sponsorship