TY - JOUR
T1 - Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa
AU - Geyer, Mark B.
AU - Radhakrishnan, Kavita
AU - Giller, Roger
AU - Umegaki, Noriko
AU - Harel, Sivan
AU - Kiuru, Maija
AU - Morel, Kimberly D.
AU - Leboeuf, Nicole
AU - Kandel, Jessica
AU - Bruckner, Anna
AU - Fabricatore, Sandra
AU - Chen, Mei
AU - Woodley, David
AU - McGrath, John
AU - Baxter-Lowe, Leeann
AU - Uitto, Jouni
AU - Christiano, Angela M.
AU - Cairo, Mitchell S.
N1 - Publisher Copyright:
© 2015 Elsevier Inc.
PY - 2015/9/1
Y1 - 2015/9/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=84940447490&partnerID=8YFLogxK
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U2 - 10.1016/j.jpeds.2015.05.051
DO - 10.1016/j.jpeds.2015.05.051
M3 - Article
C2 - 26148662
AN - SCOPUS:84940447490
SN - 0022-3476
VL - 167
SP - 765-769.e1
JO - Journal of Pediatrics
JF - Journal of Pediatrics
IS - 3
ER -