Abstract
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe blistering skin disease caused by mutations in the COL7A1 gene. These mutations lead to decreased or absent levels of collagen VII at the dermal-epidermal junction. Over the past decade, significant progress has been made in the treatment of RDEB, including the use of hematopoietic cell transplantation, but a cure has been elusive. Patients still experience life-limiting and life-threatening complications as a result of painful and debilitating wounds. The continued suffering of these patients drives the need to improve existing therapies and develop new ones. In this Review, we will discuss how recent advances in placenta-based, umbilical cord blood-based and amniotic membrane-based therapies may play a role in the both the current and future treatment of RDEB.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 786-795 |
| Number of pages | 10 |
| Journal | Cytotherapy |
| Volume | 17 |
| Issue number | 6 |
| DOIs | |
| State | Published - Jun 1 2015 |
Bibliographical note
Funding Information:We apologize to the authors whose work could not be quoted because of space constraints and thank Nancy Griggs Morgan for editorial assistance. J.T. is supported in part by R01 AR063070, R01 AR059947, DOD W81XWH, EB Research Partnership, EB Medical Research Foundation, DEBRA and the Sohana Research Fund.
Publisher Copyright:
© 2015 International Society for Cellular Therapy.
Keywords
- Bone marrow
- Epidermolysis bullosa
- Hematopoietic cell transplantation
- Induced pluripotent stem cells
- Mesenchymal stromal/stem cells
- Umbilical cord blood
