Abstract
Rapid advances in gene technology over the past decade have illuminated the molecular pathophysiology of many human diseases. Harnessing this knowledge for therapeutic purposes defines the field of gene therapy. Progresses in vector design, gene delivery, regulation of targeted gene expression and immune modulation have brought attempts at therapeutic molecular manipulation much closer to clinical use. For the field of gene therapy, the skin represents a uniquely attractive tissue site because of its accessibility and instant visualization, in addition to the large number of diseases that are principally amenable to cutaneous gene transfer. Although expression of cDNA for the treatment of autosomal recessive inherited genetic disorders has been the main advance in the field, other seminal techniques, including therapeutic trans-splicing, oligonucleotide antisense technology and RNA interference, have also emerged to target autosomal dominant mutations. Promising perspectives and challenging limitations of these approaches to cutaneous gene therapy are reviewed in this article.