Ontology highlight
ABSTRACT:
SUBMITTER: Maxwell KG
PROVIDER: S-EPMC7233417 | biostudies-literature | 2020 Apr
REPOSITORIES: biostudies-literature
Science translational medicine 20200401 540
Differentiation of insulin-producing pancreatic β cells from induced pluripotent stem cells (iPSCs) derived from patients with diabetes promises to provide autologous cells for diabetes cell replacement therapy. However, current approaches produce patient iPSC-derived β (SC-β) cells with poor function in vitro and in vivo. Here, we used CRISPR-Cas9 to correct a diabetes-causing pathogenic variant in Wolfram syndrome 1 (<i>WFS1</i>) in iPSCs derived from a patient with Wolfram syndrome (WS). Afte ...[more]