title: One goal, different strategies--molecular and cellular approaches for the treatment of inherited skin fragility disorders.
abstract: Epidermolysis bullosa (EB) is a heterogeneous group of inherited diseases characterized by the formation of blisters in the skin and mucosa. There is no cure or effective treatment for these potentially severe and fatal diseases. Over the past few years, several reports have proposed different molecular strategies as new therapeutic options for the management of EB. From classical vector-based gene therapy to cell-based strategies such as systemic application of bone marrow stem cells or local application of fibroblasts, a broad range of molecular approaches have been explored. This array also includes novel methods, such as protein replacement therapy, gene silencing and the use of induced pluripotent stem cells (iPCs). In this review, we summarize current concepts of how inherited blistering diseases might be treated in the future and discuss the opportunities, promises, concerns and risks of these innovative approaches.
authors.affiliation: Department of Dermatology, Eberhard Karls University, Tübingen, Germany.
authors.forename: Christian
authors.initials: C
authors.lastname: Hünefeld
authors.forename: Markus
authors.initials: M
authors.lastname: Mezger
authors.forename: Johannes S
authors.initials: JS
authors.lastname: Kern
authors.forename: Alexander
authors.initials: A
authors.lastname: Nyström
authors.forename: Leena
authors.initials: L
authors.lastname: Bruckner-Tuderman
authors.forename: Ingo
authors.initials: I
authors.lastname: Müller
authors.forename: Rupert
authors.initials: R
authors.lastname: Handgretinger
authors.forename: Martin
authors.initials: M
authors.lastname: Röcken
mesh_terms: D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D019468:Disease Management; D004820:Epidermolysis Bullosa / Q000628:therapy*; D020868:Gene Silencing; D015316:Genetic Therapy / Q000379:methods*; D006040:Goals; D006801:Humans; D033581:Stem Cell Transplantation; D016896:Treatment Outcome