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.
Keywords: bone marrow; epidermolysis bullosa; hematopoietic cell transplantation; induced pluripotent stem cells; mesenchymal stromal/stem cells; umbilical cord blood.
Copyright © 2015 International Society for Cellular Therapy. Published by Elsevier Inc. All rights reserved.