| Literature DB >> 26148662 |
Mark B Geyer1, Kavita Radhakrishnan2, Roger Giller3, Noriko Umegaki4, Sivan Harel4, Maija Kiuru5, Kimberly D Morel6, Nicole LeBoeuf7, Jessica Kandel8, Anna Bruckner9, Sandra Fabricatore10, Mei Chen11, David Woodley11, John McGrath12, LeeAnn Baxter-Lowe13, Jouni Uitto14, Angela M Christiano15, Mitchell S Cairo16.
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.Entities:
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Year: 2015 PMID: 26148662 DOI: 10.1016/j.jpeds.2015.05.051
Source DB: PubMed Journal: J Pediatr ISSN: 0022-3476 Impact factor: 4.406