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• W1664881034 abstract "In the past few years, substantial preclinical and experimental advances have been made in the treatment of the severe monogenic skin blistering disease epidermolysis bullosa (EB). Promising approaches have been developed in the fields of protein and cell therapies, including allogeneic stem cell transplantation; in addition, the application of gene therapy approaches has become reality. The first ex vivo gene therapy for a junctional EB (JEB) patient was performed in Italy more than 8 years ago and was shown to be effective. We have now continued this approach for an Austrian JEB patient. Further, clinical trials for a gene therapy treatment of recessive dystrophic EB are currently under way in the United States and in Europe. In this review, we aim to point out that sustainable correction of autologous keratinocytes by stable genomic integration of a therapeutic gene represents a realistic option for patients with EB." @default.
• W1664881034 created "2016-06-24" @default.
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• W1664881034 date "2015-01-01" @default.
• W1664881034 modified "2023-09-27" @default.
• W1664881034 title "Advances in Gene/Cell Therapy in Epidermolysis Bullosa" @default.
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• W1664881034 doi "https://doi.org/10.2302/kjm.2014-0013-re" @default.
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• W1664881034 hasPublicationYear "2015" @default.
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