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

Use of ex vivo stem cell gene therapy enables the correction of the genetic cause of a monogenetic skin disease.

The procedure and choice of gene therapy method in the course of ex vivo gene therapy of the skin are presented.

Current gene therapeutic applications focus on the addition or targeted correction of the respective gene within the genome.

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.

The selection of the gene therapy method depends on its safety profile, the target genodermatoses and the genetic mutation to correct.