Significant progress has been made over the past two decades in molecular genetics of epidermolysis bullosa (EB), a group of heritable blistering disorders, with diagnostic and prognostic implications. More recently, novel molecular approaches have been developed towards potential treatment of EB, with emphasis on gene-, protein-, and cell-based strategies. This overview highlights cell-based approaches that have recently been tested in pilot clinical trials, attesting to the potential of regenerative medicine for blistering skin diseases.