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Dennis Roop – University of Colorado: A stem cell-based therapy for patients with Epidermolysis Bullosa simplex.Dennis Roop – University of Colorado: A stem cell-based therapy for patients with Epidermolysis Bullosa simplex.

Beschrijving

2015 - Current therapy for Epidermolysis Bullosa Simplex (EBS) patients is primarily limited to wound care. The technological breakthrough that allows adult skin cells to be reprogrammed into immature induced pluripotent stem cells (iPSC) now offers the possibility of developing a permanent corrective therapy for EBS without the risk of immune rejection. Specifically, skin cells can be biopsied from a patient suffering from EBS and then “reprogrammed” into iPSCs. The iPSCs can then be grown outside the body, genetically corrected, differentiated into new skin stem cells, and administered back to the same patient as an autograft. Given its novelty, an iPSC-based therapy for EBS will require extensive pre-clinical research before it can be approved for a clinical trial by US and European regulatory agencies. The goal of this proposal is to generate the set of safety, potency and efficacy data required for approval of a clinical trial. For these studies, we will focus on patients suffering from the Dowling-Meara subtype of EBS (EBS-DM).

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