Translating a Stem Cell-Based Therapy for Epidermolysis Bullosa into the Clinic
Abstract
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 this funding period, we continued working on characterizing genetically corrected iPSC-derived skin cells and on developing an in vivo bioluminescent assay to determine potential tumorigenicity of iPSC-derived cells. In addition, we have continued working with our cGMP-compliant facility to advance our protocols toward clinical manufacturing. Specifically, we have identified the conditions to perform the purification of keratinocytes differentiated from iPSCs using the Clini MACS Prodigy. This step is important to ensure that keratinocytes that are being transplanted back to the patient are authentic and are not contaminated with partly differentiated cells. As indicated in the previous report, the initiation of many in vivo studies was delayed due to the COVID-19 pandemic and the closure of research laboratories on the University of Colorado Anschutz Medical Campus. Therefore, we have requested a no cost extension until September 2022, which will allow us to complete the goals of the original application. This request has been recently approved by the DOD.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2021
- Accession Number
- AD1158158
Entities
People
- Dennis R. Roop
Organizations
- University of Colorado Boulder