Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa.
case_report · Level V
Where this comes from
- Record sourced from PubMed, PMID 26148662.
- Also identified by DOI 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.
Medical subject headings
- Epidermolysis Bullosa Dystrophica
- Hematopoietic Stem Cell Transplantation
- Transplantation Conditioning