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Translating a Stem Cell-Based Therapy for Epidermolysis Bullosa into the Clinic

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Technical Report,15 Sep 2018,14 Sep 2019

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University of Colorado Denver United States

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The proposal develops a stem-cell based therapy for recessive dystrophic epidermolysis bullosa RDEB, 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 utilizing genetic correction of patient-specific induced pluripotent stem cells iPSCs followed by the differentiation of these corrected iPSCs into epidermal cells and fibroblasts for the generation of composite full thickness skin grafts for transplantation. During the first year, we have secured approval for our ACURO protocol to use animals ACURO Log Number PR171428.e001, approved on 10012019, and are currently waiting for the IRB approvals from our collaborating groups from Stanford and Columbia Universities to address HRPO concerns. While our human subject protocol was awaiting approval by the DOD, we focused on tasks that did not require human subject participation. Specifically, we validated antibodies necessary to accomplish the project we are also transferring our protocols for the generation of iPSCs and iPSC-derived keratinocytes into the Gates Biomanufacturing Facility GBF to implement a pilot cGMP run and we have finalized the protocol for the differentiation of iPSCs into fibroblasts. We have also assessed several matrices to generate full thickness skin equivalents for the proposed research.

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  • Medicine and Medical Research

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