Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa

BACKGROUND: 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 h...

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Autori principali: Wagner, John E., Ishida-Yamamoto, Akemi, McGrath, John A., Hordinsky, Maria, Keene, Douglas R., Riddle, Megan J., Osborn, Mark J., Lund, Troy, Dolan, Michelle, Blazar, Bruce R., Tolar, Jakub
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2010
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2967187/
https://ncbi.nlm.nih.gov/pubmed/20818854
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa0910501
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