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Stem Cell-Based Therapies for Epidermolysis Bullosa

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Technical Report,30 Sep 2012,29 Sep 2015

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Regents of the University of Colorado Aurora United States

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This application addresses the FY11 PRMRP Topic Area, Epidermolysis Bullosa, and proposes to develop stem-cell based therapies for junctional epidermolysis bullosa JEB, which is one of the most severe forms of epidermolysis bullosa EB, a group of rare inherited skin blistering diseases. To accomplish this goal, we are proposing to develop stem-cell based therapies for EB using autologous induced pluripotent stem cells iPSCs derived from skin cells harvested from the same EB patient. During the entire funding period, we successfully accomplished most of the milestones toward the development of a proof-of-concept genome editing therapeutic strategy for JEB patients proposed as a main goal of this DOD application. We adapted integration-free reprogramming to both mouse and human somatic cells. We optimized iPSC differentiation protocols toward keratinocyte and mesenchymal lineages. We are currently completing gene editing of JEB iPSCs, fulfilling the final task for the successful accomplishment of the proposed study. Our developed iPSC-based stem cell therapy can be applicable not only to JEB patients but also to patients with other inherited skin diseases, as well as veterans with chronic wounds.

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