Ulrich Koller1. 1. Universitätsklinik für Dermatologie und Allergologie, EB-Haus Austria, Universitätsklinikum der Paracelsus Medizinischen Privatuniversität Salzburg, Müllner Hauptstraße 48, 5020, Salzburg, Österreich. u.koller@salk.at.
Abstract
BACKGROUND: Use of ex vivo stem cell gene therapy enables the correction of the genetic cause of a monogenetic skin disease. OBJECTIVES: The procedure and choice of gene therapy method in the course of ex vivo gene therapy of the skin are presented. MATERIALS AND METHODS: Current gene therapeutic applications focus on the addition or targeted correction of the respective gene within the genome. RESULTS: So far, gene replacement therapy has been successfully used in patients suffering from the blistering skin disease epidermolysis bullosa. Designer nuclease-based gene therapy approaches are at the preclinical stage. CONCLUSIONS: The selection of the gene therapy method depends on its safety profile, the target genodermatoses and the genetic mutation to correct.
BACKGROUND: Use of ex vivo stem cell gene therapy enables the correction of the genetic cause of a monogenetic skin disease. OBJECTIVES: The procedure and choice of gene therapy method in the course of ex vivo gene therapy of the skin are presented. MATERIALS AND METHODS: Current gene therapeutic applications focus on the addition or targeted correction of the respective gene within the genome. RESULTS: So far, gene replacement therapy has been successfully used in patients suffering from the blistering skin disease epidermolysis bullosa. Designer nuclease-based gene therapy approaches are at the preclinical stage. CONCLUSIONS: The selection of the gene therapy method depends on its safety profile, the target genodermatoses and the genetic mutation to correct.
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