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Can people genetically unable to synthesize a protein use it if injected? Specifically, can injection of type VII collagen (C-VII) help patients with recessive dystrophic epidermolysis bullosa (RDEB) who lack this protein? Previously, these researchers injected C-VII in a mouse model of RDEB, where, perhaps surprisingly, it was promptly incorporated into the basement membrane zone. Injections into human, organotypic, constructed RDEB-equivalent skin lacking C-VII that had been transplanted onto immunocompromised mice were similarly successful.
Systemic protein therapy has been primarily employed in systemic diseases such as hemophilia or lysosomal storage disorders. Woodley and others wondered if intravenously injected recombinant C-VII (rC-…