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

研究成果: Article同行評審

26 引文 斯高帕斯(Scopus)

摘要

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.

原文English
頁(從 - 到)765-769.e1
期刊Journal of Pediatrics
167
發行號3
DOIs
出版狀態Published - 2015 9月 1

All Science Journal Classification (ASJC) codes

  • 兒科、圍產兒和兒童健康

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