Article
Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa.
The Journal of pediatrics - 1 Sept 2015
Geyer Mark B, Radhakrishnan Kavita, Giller Roger, Umegaki Noriko, Harel Sivan, Kiuru Maija, Morel Kimberly D, LeBoeuf Nicole, Kandel Jessica, Bruckner Anna, Fabricatore Sandra, Chen Mei, Woodley David, McGrath John, Baxter-Lowe LeeAnn, Uitto Jouni, Christiano Angela M, Cairo Mitchell S
Abstract excerpt
Recessive dystrophic epidermolysis bullosa is a severe, incurable, inherited blistering disease caused by COL7A1 mutations. Emerging evidence suggests hematopoietic progenitor cells (HPCs) can be reprogrammed into skin; HPC-derived cells can restore COL7 expression in COL7-deficient mice. We report two children with recessive dystrophic epidermolysis bullosa treated with reduced-toxicity conditioning and...
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