Testing the Therapeutic Potential of Induced Pluripotent Stem Cells (iPSC) for Epidermolysis Bullosa Simplex (EBS) (Roop 1)Completed
|Prof Dennis Roop
|University of Colorado Denver, Denver, USA
|Start date / Duration
|01. Jul 2010 / 36 months
|Funder(s) / Co-Funder(s)
|DEBRA Austria, MSAP/EBEP Recommended
Short lay summary
Besides symptomatic care, no effective therapeutic treatment is available for EBS and other forms of EB. Therefore, a stem cell/gene therapy based approach is the only option for a permanent corrective therapy for these patients. The discovery that adult human skin cells could be reprogrammed into an embryonic stem cell (ESC)-like state has had a dramatic effect on the fields of stem cell biology and regenerative medicine. The therapeutic potential of such induced pluripotent stem cells (iPSC) for tissue repair and regeneration is enormous. This procedure not only eliminates ethical concerns associated with generating ESCs from fertilized human embryos, but it also enables the delivery of truly personalized medical treatment since the iPSC would be generated from the same individual in need of treatment. Furthermore, the therapeutic use of cells derived from a patient’s own iPSC would potentially avoid the complication of immune rejection, which might occur if cells were derived from ESCs.
However, before iPSC can be safely used in the clinic, several obstacles must be overcome. We have proposed a comprehensive set of experiments designed to address these obstacles. If we are successful in addressing the safety issues associated with using patient-specific iPSC for EBS, then this would further expand the therapeutic potential of iPSC for the treatment of other forms of EB.