Current Advances in Gene Therapy for the Treatment of Genodermatoses

Long, Heather A., McMillan, James, Qiao, Hongjiang, Akiyama, Masashi and Shimizu, Hiroshi (2009) Current Advances in Gene Therapy for the Treatment of Genodermatoses. Current Gene Therapy, 9 6: 487-494. doi:10.2174/156652309790031139

Author Long, Heather A.
McMillan, James
Qiao, Hongjiang
Akiyama, Masashi
Shimizu, Hiroshi
Title Current Advances in Gene Therapy for the Treatment of Genodermatoses
Journal name Current Gene Therapy   Check publisher's open access policy
ISSN 1566-5232
Publication date 2009-12
Year available 2009
Sub-type Critical review of research, literature review, critical commentary
DOI 10.2174/156652309790031139
Open Access Status
Volume 9
Issue 6
Start page 487
End page 494
Total pages 8
Place of publication Netherlands
Publisher Bentham Science
Language eng
Subject 920117 Skin and Related Disorders
110304 Dermatology
110311 Medical Genetics (excl. Cancer Genetics)
Formatted abstract
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.
Q-Index Code C1
Q-Index Status Provisional Code
Institutional Status Unknown

Document type: Journal Article
Sub-type: Critical review of research, literature review, critical commentary
Collection: School of Medicine Publications
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Citation counts: TR Web of Science Citation Count  Cited 5 times in Thomson Reuters Web of Science Article | Citations
Scopus Citation Count Cited 6 times in Scopus Article | Citations
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Created: Wed, 17 Mar 2010, 18:57:15 EST by Amanda Jones on behalf of Paediatrics & Child Health - RBWH