Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem Cells
Recessive dystrophic epidermolysis bullosa is a severe skin fragility disease caused by loss of functional type VII collagen at the dermal-epidermal junction.A frameshift mutation in exon 80 of COL7A1 gene, c.6527insC, is highly prevalent in Eye mask the Spanish patient population.We have implemented gene-editing strategies for COL7A1 frame restora