Gene therapy prevents cancer in mice with xeroderma pigmentosum

NEW YORK (Reuters Health) - Delivery of the normal xeroderma pigmentosum A (XPA) gene to mice with xeroderma pigmentosum can prevent the development of squamous cell carcinoma, according to a report in the December 6th early edition of the Proceeding of the National Academy of Sciences.

The inability to effectively repair their DNA places xeroderma pigmentosum patients at increased risk of skin cancer. The disease typically stems from an inactivating mutation in one of seven xeroderma pigmentosum genes.

Dr. Carlos F. M. Menck, from Universidade de Sao Paulo in Brazil, and colleagues used a recombinant adenovirus to deliver a normal copy of the XPA gene to xeroderma pigmentosum-mutant mice, an animal model of xeroderma pigmentosum that closely mimics the human phenotype. The virus was administered with a subcutaneous injection.

Like their human counterparts, these mice are exquisitely sensitive to UV radiation-induced skin tumors, the researchers point out.

Treatment with the gene therapy led to the expression of the XPA protein in skin cells and prevented the development of skin cancer.

“The success of gene delivery to skin cells and genetic correction in Xpa-mutant mice provides an exciting technology to investigate DNA repair functions in vivo,” the authors note. “These results also motivate continued searches for improved methodologies to optimize gene therapy protocols with the aim of offering an improved quality of life for xeroderma pigmentosum patients.”

Source: Proc Natl Acad Sci USA 2004. [ Google search on this article ]

MeSH Headings: Biological Therapy : Genetic Engineering : Genetic Techniques : Investigative Techniques : Therapeutics : Gene Therapy : Analytical, Diagnostic and Therapeutic Techniques and Equipment

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