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

doi:10.1016/j.jpeds.2015.05.051

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

皮膚科学

研究成果: Article › 査読

28 被引用数 (Scopus)

抄録

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.

本文言語	English
ページ（範囲）	765-769.e1
ジャーナル	Journal of Pediatrics
巻	167
号	3
DOI	https://doi.org/10.1016/j.jpeds.2015.05.051
出版ステータス	Published - 2015 9月 1

ASJC Scopus subject areas

小児科学、周産期医学および子どもの健康

文献へのアクセス

10.1016/j.jpeds.2015.05.051

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引用スタイル

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

Geyer, MB, Radhakrishnan, K, Giller, R, Umegaki, N, Harel, S, Kiuru, M, Morel, KD, Leboeuf, N, Kandel, J, Bruckner, A, Fabricatore, S, Chen, M, Woodley, D, McGrath, J, Baxter-Lowe, L, Uitto, J, Christiano, AM & Cairo, MS 2015, 'Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa', Journal of Pediatrics, vol. 167, no. 3, pp. 765-769.e1. https://doi.org/10.1016/j.jpeds.2015.05.051

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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.",

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