8th International Wolfram Symposium Presentation Prof Catherine Verfaillie – Stem Cell Institute, KU Leuven, Belgium
8th International Wolfram Symposium Presentation Prof Catherine Verfaillie – Stem Cell Institute, KU Leuven, Belgium
iPSC-based modeling of Wolfram Syndrome and therapeutic genome editing approaches
Points noted:
• Developed iPSC-based models of different cell types – neural cells, ganglion, astrocytes, and blood brain barrier endothelial cells.
• These cell lines can be shared with other WS researchers who would like to use them.
• Further evaluate (e.g. myelination, stain for different markers) and explore changes over time. Also compare with WS-derived models to explore differences.
• Base editing conducted using this system – look to see if / how this can be delivered in vivo.
• Translate findings to mouse model – exchange with Fumi (Dr Urano) mouse model with current iPSC system.