Review

Gene Therapy (2006) 13, 1555–1563. doi:10.1038/sj.gt.3302855; published online 7 September 2006

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

U R Hengge1

1Department of Dermatology, Heinrich-Heine-University, Duesseldorf, Germany

Correspondence: Professor UR Hengge, Department of Dermatology, Heinrich-Heine-University, Moorenstrasse 5, D-40225 Duesseldorf, Germany. E-mail: ulrich.hengge@uni-duesseldorf.de

Received 29 November 2005; Revised 12 July 2006; Accepted 22 July 2006; Published online 7 September 2006.

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Abstract

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.

Keywords:

inherited blistering skin diseases, skin gene therapy, DNA vaccination, internalization pathways, trafficking, immune response

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