Systemic protein therapy for recessive dystrophic epidermolysis bullosa: how far are we from clinical translation?
In this issue, Woodley et al. report restoration of anchoring fibril formation and dermal-epidermal adherence in a murine model of recessive dystrophic epidermolysis bullosa (RDEB) by intravenous injection of recombinant human type VII collagen. This work follows a previous report by the same group of the surprising capability of intradermally injected type VII collagen protein to reverse RDEB, and it opens new therapeutic avenues.