CRISPR/Cas9 Based Non-Viral Gene Editing Therapy for Topical Treatment of Recessive Dystrophic Epidermolysis Bullosa

CRISPR/Cas9 plasmids delivered by the selcted highly branched poly(β amino ester)s achieved efficient targeted deletion and restored bulk C7 production in recessive dystrophic epidermolysis bullosa patient keratinocyte polyclones.