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Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa
Institution:1. EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria;2. Institute for Transfusion Medicine and Gene Therapy, Medical Center – University of Freiburg, Freiburg, Germany;3. Center for Chronic Immunodeficiency, Medical Center – University of Freiburg, Freiburg, Germany;4. Cell Therapy Institute, SCI-TReCS, Paracelsus Medical University, Salzburg, Austria;5. Epithelial Biomedicine Division, CIEMAT-CIBERER, Department of Bioengineering, UC3M, Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz, 28040 Madrid, Spain;6. Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, 5020 Salzburg, Austria;7. Faculty of Medicine, University of Freiburg, Freiburg, Germany
Abstract:
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Keywords:junctional epidermolysis bullosa  JEB  paired nicking  gene reframing  CRISPR-Cas9  next-generation sequencing  skin equivalents
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