Heather A. Long, James R. McMillan, Hongjiang Qiao, Masashi Akiyama and Hiroshi Shimizu Pages 487 - 494 ( 8 )
Gene therapy provides the possibility of long term treatment for the severest of congenital disorders. In this review we will examine the recent advances in gene therapy for genodermatoses. Congenital diseases of the skin exhibit a wide range of severity and underlying causes and there are many possible therapeutic avenues. Gene therapy approaches can follow three paths-in vivo, ex vivo and fetal gene therapy, though the later is currently theoretical only it can provide potential results for even the most severe congenital diseases. All approaches utilize the many different vector systems available, including viral and the emerging use of non- viral integrating vectors. In addition, the use of RNAi based techniques to prevent dominant mutant protein expression has been explored as a therapy for specific dominant disorders such as keratin mutation disorders. Progress has been rapid in the past few years with some initial successful clinical trials reported. However, there are still some issues surrounding long term expression, transgene sustainability and safety issues that need to be addressed to further shift from experimental to clinically therapeutic applications. With the continuing development, merger and refinement of existing techniques there is an ever increasing likelihood of gene therapies becoming available for the more severe genodermatoses within the next decade or shortly thereafter., Current, Advances, in, Gene, Therapy, for, the, Treatment, of, Genodermatoses
Department of Dermatology, Hokkaido University Graduate School of Medicine, N15 W7, Kita-ku, Sapporo 060-8638, Japan.