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Nat Med. 2004 Jul;10(7):693-5. Epub 2004 Jun 13.

Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa.

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Department of Medicine, Division of Dermatology, University of Southern California, CRL 204, 1303 Mission Road, Los Angeles, California 90033, USA.


Dystrophic epidermolysis bullosa (DEB) is a family of inherited mechano-bullous disorders that are caused by mutations in the type VII collagen gene and for which ex vivo gene therapy has been considered. To develop a simpler approach for treating DEB, we evaluated the feasibility of protein-based therapy by intradermally injecting human recombinant type VII collagen into mouse skin and a DEB human skin equivalent transplanted onto mice. The injected collagen localized to the basement membrane zone of both types of tissues, was organized into human anchoring fibril structures and reversed the features of DEB disease in the DEB skin equivalent.

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