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Significant progress has been made in corrective gene therapy of inherited skin diseases. This includes advances in vector technology, targeted gene expression, gene replacement, and the availability of appropriate animal models for a variety of candidate diseases. In addition, an increased understanding of the uptake and trafficking mechanisms inside keratinocytes has evolved. Topical application facilitates DNA vaccination through the skin, albeit clinical benefits have not yet materialized. However, the translation into clinical trials has only been partially mastered. The latter and the control of immune responses represent challenges for the research community.

Gene therapy progress and prospects: the skin – easily accessible, but still far away