The skin fragility disorder recessive dystrophic epidermolysis bullosa (RDEB) results from mutations in COL7A1 leading to reduced or absent type VII collagen (C7) and defective anchoring fibrils at the dermal-epidermal junction (DEJ)(Fine et al., 2014). Currently, there is no cure and most individuals develop life-shortening squamous cell carcinomas (Fine and Mellerio, 2009). RDEB also has a major health economic burden; wound dressings for a 10-year old child can cost $680 per day (Kirkorian et al., 2014), which equates to> $250,000 annually.

Reported clinical trials of cell-based therapies for RDEB comprise intradermal allogeneic fibroblasts (Petrof et al., 2013; Venugopal et al., 2013), and bone marrow transplantation (Wagner et al., 2010), as well as intradermal bone marrow-derived mesenchymal stromal cells (BM-MSCs)(Conget et al., 2010), and intravenous BM-MSCs in RDEB adults (El-Darouti et al., 2013; abstract only). Ex vivo COL7A1 keratinocyte gene therapy is also being evaluated (Siprashvili et al., 2014).