pubmed:abstractText |
Recessive dystrophic epidermolysis bullosa is an incurable, often fatal mucocutaneous blistering disease caused by mutations in COL7A1, the gene encoding type VII collagen (C7). On the basis of preclinical data showing biochemical correction and prolonged survival in col7 ?/? mice, we hypothesized that allogeneic marrow contains stem cells capable of ameliorating the manifestations of recessive dystrophic epidermolysis bullosa in humans.
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pubmed:affiliation |
Blood and Marrow Transplant Program, Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA. wagne002@umn.edu
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pubmed:publicationType |
Journal Article,
Research Support, Non-U.S. Gov't,
Clinical Trial, Phase II,
Clinical Trial, Phase I,
Research Support, N.I.H., Extramural
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