基于胎盘的疗法治疗大疱性表皮松解症。
Placenta-based therapies for the treatment of epidermolysis bullosa.
作者信息
Nevala-Plagemann Christopher, Lee Catherine, Tolar Jakub
机构信息
Stem Cell Institute and Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
Stem Cell Institute and Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
出版信息
Cytotherapy. 2015 Jun;17(6):786-795. doi: 10.1016/j.jcyt.2015.03.006. Epub 2015 Mar 18.
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.
摘要
隐性营养不良性大疱性表皮松解症（RDEB）是一种由COL7A1基因突变引起的严重皮肤水疱病。这些突变导致真皮-表皮交界处的VII型胶原蛋白水平降低或缺失。在过去十年中，RDEB的治疗取得了重大进展，包括使用造血细胞移植，但仍未找到治愈方法。由于伤口疼痛和虚弱，患者仍然面临危及生命和限制生命的并发症。这些患者的持续痛苦促使人们需要改进现有疗法并开发新疗法。在本综述中，我们将讨论基于胎盘、脐带血和羊膜的疗法的最新进展如何可能在RDEB的当前和未来治疗中发挥作用。
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