Inducible Pluripotent Stem Cells
The availability of human embryonic stem cells (hESC) and inducible, pluripotent stem cell (iPSC)-technology through the University of Rochester Stem Cell and Regenerative Medicine Institute, and the Upstate cGMP Facility, provide unique opportunities to establish new in vitro models of CNS disease, and to develop new cell therapies.
Using patient-derived cells to generate inducible, pluripotent stem cells (iPSCs), we have now developed a Disease in a Dish model of VWM. In collaboration with the Department of Neurology at Massachusetts General Hospital, (Harvard), we will use these in vitro cell models to further study the cell biological basis of VWM-disease and to screen for candidate therapeutic compounds.