Treatment of hereditary epidermolysis bullosa: Updates and future prospects

Research output: Contribution to journalArticle

23 Citations (Scopus)

Abstract

Epidermolysis bullosa (EB) represents a group of inherited blistering skin diseases, some forms of which are associated with considerable morbidity and increased mortality. Notably, in recessive dystrophic EB there can be extensive muco-cutaneous fragility and disease complications such as scars, contractures, anemia, malnutrition, and malignancy. Currently, there is no effective therapy or cure for EB. Over the last decade, however, a number of important advances have been made that are bringing new treatments closer to the clinic, including gene therapy, protein replacement therapy, cell therapies [allogeneic fibroblasts, mesenchymal stromal cells (MSCs), bone marrow stem cell transplantation, culturing/grafting revertant mosaic keratinocytes], gene editing/engineering, and clinical application of inducible pluripotent stem cells. Although a cure for EB still remains elusive, recent data on animal models and initial human clinical trials have raised the expectations of patients, clinicians, and researchers that disease modification and improved quality of life are feasible goals. Furthermore, the lessons learned in treating EB are likely to have significant implications for improving the management of other genetic diseases.

Original languageEnglish
Pages (from-to)1-6
Number of pages6
JournalAmerican Journal of Clinical Dermatology
Volume15
Issue number1
DOIs
Publication statusPublished - 2014 Feb 1

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Epidermolysis Bullosa
Skin Diseases
Epidermolysis Bullosa Dystrophica
Biomedical Engineering
Pluripotent Stem Cells
Inborn Genetic Diseases
Stem Cell Transplantation
Contracture
Therapeutics
Cell- and Tissue-Based Therapy
Bone Marrow Transplantation
Mesenchymal Stromal Cells
Keratinocytes
Malnutrition
Genetic Therapy
Cicatrix
Anemia
Animal Models
Fibroblasts
Quality of Life

All Science Journal Classification (ASJC) codes

  • Dermatology

Cite this

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abstract = "Epidermolysis bullosa (EB) represents a group of inherited blistering skin diseases, some forms of which are associated with considerable morbidity and increased mortality. Notably, in recessive dystrophic EB there can be extensive muco-cutaneous fragility and disease complications such as scars, contractures, anemia, malnutrition, and malignancy. Currently, there is no effective therapy or cure for EB. Over the last decade, however, a number of important advances have been made that are bringing new treatments closer to the clinic, including gene therapy, protein replacement therapy, cell therapies [allogeneic fibroblasts, mesenchymal stromal cells (MSCs), bone marrow stem cell transplantation, culturing/grafting revertant mosaic keratinocytes], gene editing/engineering, and clinical application of inducible pluripotent stem cells. Although a cure for EB still remains elusive, recent data on animal models and initial human clinical trials have raised the expectations of patients, clinicians, and researchers that disease modification and improved quality of life are feasible goals. Furthermore, the lessons learned in treating EB are likely to have significant implications for improving the management of other genetic diseases.",
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Treatment of hereditary epidermolysis bullosa : Updates and future prospects. / Hsu, Chao Kai; Wang, Sheng Pei; Lee, Julia Yu Yun; McGrath, John A.

In: American Journal of Clinical Dermatology, Vol. 15, No. 1, 01.02.2014, p. 1-6.

Research output: Contribution to journalArticle

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