Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic
Mark B Geyer1, Kavita Radhakrishnan2, Roger Giller3
1Department of Medicine (Hematology and Medical Oncology), Memorial Sloan-Kettering Cancer Center, New York, NY.
The Journal of Pediatrics
|July 8, 2015
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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