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Nat Commun. 2016 May 10;7:11463. doi: 10.1038/ncomms11463.

Generation of stem cell-derived β-cells from patients with type 1 diabetes.

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Division of Endocrinology, Metabolism and Lipid Research, Washington University School of Medicine, Campus Box 8127, 660 South Euclid Avenue, St Louis, Missouri 63110, USA.
Department of Biomedical Engineering, Washington University in St Louis, 1 Brookings Drive, St Louis, Missouri 63130, USA.
Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute, Harvard University 7 Divinity Avenue, Cambridge, Massachusetts 02138, USA.
Case Western Reserve University School of Medicine, 2109 Adelbert Road, Cleveland, Ohio 44106, USA.
Semma Therapeutics, Inc. 450 Kendall Street, Suite 2B Cambridge, Massachusetts 02142, USA.


We recently reported the scalable in vitro production of functional stem cell-derived β-cells (SC-β cells). Here we extend this approach to generate the first SC-β cells from type 1 diabetic patients (T1D). β-cells are destroyed during T1D disease progression, making it difficult to extensively study them in the past. These T1D SC-β cells express β-cell markers, respond to glucose both in vitro and in vivo, prevent alloxan-induced diabetes in mice and respond to anti-diabetic drugs. Furthermore, we use an in vitro disease model to demonstrate the cells respond to different forms of β-cell stress. Using these assays, we find no major differences in T1D SC-β cells compared with SC-β cells derived from non-diabetic patients. These results show that T1D SC-β cells could potentially be used for the treatment of diabetes, drug screening and the study of β-cell biology.

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