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One goal, different strategies – molecular and cellular approaches for the treatment of inherited skin fragility disorders
Author(s) -
Hünefeld Christian,
Mezger Markus,
Kern Johannes S.,
Nyström Alexander,
BrucknerTuderman Leena,
Müller Ingo,
Handgretinger Rupert,
Röcken Martin
Publication year - 2013
Publication title -
experimental dermatology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.108
H-Index - 96
eISSN - 1600-0625
pISSN - 0906-6705
DOI - 10.1111/exd.12084
Subject(s) - epidermolysis bullosa , genetic enhancement , medicine , gene silencing , stem cell , induced pluripotent stem cell , bioinformatics , biology , gene , dermatology , embryonic stem cell , genetics
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 (i PC s). 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.