One goal, different strategies - molecular and cellular approaches for the treatment of inherited skin fragility disorders

Christian Hünefeld, Markus Mezger, Johannes S. Kern, Alexander Nyström, Leena Bruckner-Tuderman, Ingo Müller, Rupert Handgretinger, Martin Röcken

Research output: Contribution to journalReview ArticleResearchpeer-review

15 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)162-167
Number of pages6
JournalExperimental Dermatology
Issue number3
Publication statusPublished - Mar 2013
Externally publishedYes


  • Blistering disease
  • Epidermolysis bullosa
  • Fibroblast and protein therapy
  • Gene therapy
  • Mesenchymal stromal cell
  • Stem cell transplantation

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