Progress and prospects of skin gene therapy: a ten year history

Significant progress has been made in corrective gene therapy of the skin in the last decade. This includes advances in vector technology, targeted gene expression, gene replacement, gene correction, and the availability of appropriate animal models for a variety of candidate diseases. While non-viral integration of large genes such as essential basement membrane proteins has been mastered, new challenges such as the control of immune responses lie ahead of the research community until skin gene therapy will become clinical reality. Among the first skin diseases patients with junctional epidermolysis bullosa and xeroderma pigmentosum have entered clinical trials.