Highly Efficient Ex Vivo Correction of COL7A1 through RNP-Based CRISPR/Cas9 and Homology-Directed Repair to Treat Recessive Dystrophic Epidermolysis Bullosa

Investigators achieved highly efficient COL7A1 editing in primary recessive dystrophic epidermolysis bullosa keratinocytes and fibroblasts from two patients homozygous for the c.6508C>T mutation.