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

Department of Dermatology

Research output: Contribution to journal › Article › peer-review

21 Citations (Scopus)

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.

Original language	English
Pages (from-to)	765-769.e1
Journal	Journal of Pediatrics
Volume	167
Issue number	3
DOIs	https://doi.org/10.1016/j.jpeds.2015.05.051
Publication status	Published - 2015 Sept 1

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health

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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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title = "Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa",

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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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

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AU - Uitto, Jouni

AU - Christiano, Angela M.

AU - Cairo, Mitchell S.

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Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa

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