[Ex vivo stem cell gene therapy of the skin : Ready for clinical use?].

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.