Gene-edited human stem cell–derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice-Reference-Cited by-同舟云学术

Gene-edited human stem cell–derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice

Published:2020-04-22 Issue:540 Volume:12 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Maxwell Kristina G.¹²^ORCID,Augsornworawat Punn¹²^ORCID,Velazco-Cruz Leonardo¹^ORCID,Kim Michelle H.¹^ORCID,Asada Rie¹^ORCID,Hogrebe Nathaniel J.¹^ORCID,Morikawa Shuntaro¹^ORCID,Urano Fumihiko¹³^ORCID,Millman Jeffrey R.¹²^ORCID

Affiliation:

1. Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.

2. Department of Biomedical Engineering, Washington University in St. Louis, 1 Brookings Drive, St. Louis, MO 63130, USA.

3. Department of Pathology and Immunology, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.

Abstract

Patient stem cell–derived β cells corrected for a diabetes-causing variant in WFS1 restore glucose homeostasis when transplanted into diabetic mice.

Funder

National Institutes of Health

National Institute of General Medical Sciences

National Institute of Diabetes and Digestive and Kidney Diseases

National Center for Advancing Translational Sciences

Unravel Wolfram Syndrome Fund

Silberman Fund

Stowe Fund