Journal
LANCET
Volume 382, Issue 9899, Pages 1214-1223Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/S0140-6736(13)61897-8
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Funding
- National Institutes of Health [R01 AR063070, R01AR059947]
- US Department of Defense [W81XWH-12-1-0609]
- Epidermolysis Bullosa Research Fund
- Jackson Gabriel Silver Foundation
- DEBRA
- University of Minnesota Academic Health Center
- Pioneering Unique Cures for Kids Foundation
- Liao Family Fund
- Sarah Moreland Fund
- Children's Cancer Research Fund (Minneapolis, MN, USA)
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Contrary to the prevailing professional opinion of the past few decades, recent experimental and clinical data support the fact that protein replacement therapy by allogeneic blood and marrow transplantation is not limited to freely diffusible molecules such as enzymes, but also large structural proteins such as collagens. A prime example is the cross-correction of type VII collagen deficiency in generalised severe recessive dystrophic epidermolysis bullosa, in which blood and marrow transplantation can attenuate the mucocutaneous manifestations of the disease and improve patients' quality of life. Although allogeneic blood and marrow transplantation can improve the integrity of the skin and mucous membranes, today's accomplishments are only the first steps on the long pathway to cure. Future strategies will be built on the lessons learned from these first transplant studies.
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