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

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One goal, different strategies--molecular and cellular approaches for the treatment of inherited skin fragility disorders. / Hünefeld, Christian; Mezger, Markus; Kern, Johannes S; Nyström, Alexander; Bruckner-Tuderman, Leena; Müller, Ingo; Handgretinger, Rupert; Röcken, Martin.

in: EXP DERMATOL, Jahrgang 22, Nr. 3, 01.03.2013, S. 162-7.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung

Harvard

Hünefeld, C, Mezger, M, Kern, JS, Nyström, A, Bruckner-Tuderman, L, Müller, I, Handgretinger, R & Röcken, M 2013, 'One goal, different strategies--molecular and cellular approaches for the treatment of inherited skin fragility disorders', EXP DERMATOL, Jg. 22, Nr. 3, S. 162-7. https://doi.org/10.1111/exd.12084

APA

Hünefeld, C., Mezger, M., Kern, J. S., Nyström, A., Bruckner-Tuderman, L., Müller, I., Handgretinger, R., & Röcken, M. (2013). One goal, different strategies--molecular and cellular approaches for the treatment of inherited skin fragility disorders. EXP DERMATOL, 22(3), 162-7. https://doi.org/10.1111/exd.12084

Vancouver

Hünefeld C, Mezger M, Kern JS, Nyström A, Bruckner-Tuderman L, Müller I et al. One goal, different strategies--molecular and cellular approaches for the treatment of inherited skin fragility disorders. EXP DERMATOL. 2013 Mär 1;22(3):162-7. https://doi.org/10.1111/exd.12084

Bibtex

@article{00cce9673c7a44558ba755ba62a46587,
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.",
keywords = "Cell- and Tissue-Based Therapy, Disease Management, Epidermolysis Bullosa, Gene Silencing, Genetic Therapy, Goals, Humans, Stem Cell Transplantation, Treatment Outcome",
author = "Christian H{\"u}nefeld and Markus Mezger and Kern, {Johannes S} and Alexander Nystr{\"o}m and Leena Bruckner-Tuderman and Ingo M{\"u}ller and Rupert Handgretinger and Martin R{\"o}cken",
note = "{\textcopyright} 2013 John Wiley & Sons A/S.",
year = "2013",
month = mar,
day = "1",
doi = "10.1111/exd.12084",
language = "English",
volume = "22",
pages = "162--7",
journal = "EXP DERMATOL",
issn = "0906-6705",
publisher = "Wiley-Blackwell",
number = "3",

}

RIS

TY - JOUR

T1 - One goal, different strategies--molecular and cellular approaches for the treatment of inherited skin fragility disorders

AU - Hünefeld, Christian

AU - Mezger, Markus

AU - Kern, Johannes S

AU - Nyström, Alexander

AU - Bruckner-Tuderman, Leena

AU - Müller, Ingo

AU - Handgretinger, Rupert

AU - Röcken, Martin

N1 - © 2013 John Wiley & Sons A/S.

PY - 2013/3/1

Y1 - 2013/3/1

N2 - 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.

AB - 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.

KW - Cell- and Tissue-Based Therapy

KW - Disease Management

KW - Epidermolysis Bullosa

KW - Gene Silencing

KW - Genetic Therapy

KW - Goals

KW - Humans

KW - Stem Cell Transplantation

KW - Treatment Outcome

U2 - 10.1111/exd.12084

DO - 10.1111/exd.12084

M3 - SCORING: Journal article

C2 - 23489418

VL - 22

SP - 162

EP - 167

JO - EXP DERMATOL

JF - EXP DERMATOL

SN - 0906-6705

IS - 3

ER -