Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa

Mark B. Geyer, Kavita Radhakrishnan, Roger Giller, Noriko Umegaki, Sivan Harel, Maija Kiuru, Kimberly D. Morel, Nicole Leboeuf, Jessica Kandel, Anna Bruckner, Sandra Fabricatore, Mei Chen, David Woodley, John McGrath, Leeann Baxter-Lowe, Jouni Uitto, Angela M. Christiano, Mitchell S. Cairo

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Abstract

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 HLA-matched HPC transplantation.

Original languageEnglish
Pages (from-to)765-769.e1
JournalJournal of Pediatrics
Volume167
Issue number3
DOIs
Publication statusPublished - 2015 Sep 1
Externally publishedYes

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ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

Geyer, M. B., Radhakrishnan, K., Giller, R., Umegaki, N., Harel, S., Kiuru, M., Morel, K. D., Leboeuf, N., Kandel, J., Bruckner, A., Fabricatore, S., Chen, M., Woodley, D., McGrath, J., Baxter-Lowe, L., Uitto, J., Christiano, A. M., & Cairo, M. S. (2015). Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa. Journal of Pediatrics, 167(3), 765-769.e1. https://doi.org/10.1016/j.jpeds.2015.05.051