Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
6221034 | The Journal of Pediatrics | 2015 | 6 Pages |
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.
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Authors
Mark B. MD, Kavita MD, Roger MD, Noriko MD, PhD, Sivan PhD, Maija MD, PhD, Kimberly D. MD, Nicole MD, Jessica MD, Anna MD, Sandra RN, MSN, Mei PhD, David MD, John MD, LeeAnn PhD, Jouni MD, PhD, Angela M. PhD, Mitchell S. MD,