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

28 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

Access to Document

10.1016/j.jpeds.2015.05.051

Cite this

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

@article{574dc7e490364509aec0bc708ed1a6e2,

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.",

author = "Geyer, \{Mark B.\} and Kavita Radhakrishnan and Roger Giller and Noriko Umegaki and Sivan Harel and Maija Kiuru and Morel, \{Kimberly D.\} and Nicole Leboeuf and Jessica Kandel and Anna Bruckner and Sandra Fabricatore and Mei Chen and David Woodley and John McGrath and Leeann Baxter-Lowe and Jouni Uitto and Christiano, \{Angela M.\} and Cairo, \{Mitchell S.\}",

note = "Publisher Copyright: {\textcopyright} 2015 Elsevier Inc.",

year = "2015",

month = sep,

day = "1",

doi = "10.1016/j.jpeds.2015.05.051",

language = "English",

volume = "167",

pages = "765--769.e1",

journal = "Journal of Pediatrics",

issn = "0022-3476",

publisher = "Elsevier Inc.",

number = "3",

}

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.

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 - https://www.scopus.com/pages/publications/84940447490

UR - https://www.scopus.com/pages/publications/84940447490#tab=citedBy

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 -

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

Abstract

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this