Genes transduction in skin cells: Preventing cancer in xeroderma pigmentosum mice

Maria Carolina N Marchetto, Alysson R. Muotri, Dennis K. Burns, Errol C. Friedberg, Carlos F M Menck

Research output: Contribution to journalArticlepeer-review

45 Scopus citations


UV radiation is the most common risk factor for skin cancer. Patients with the autosomal recessive DNA repair disorder xeroderma pigmentosum (XP) suffer high incidence of skin cancer after sunlight exposure. XP-mutant mice are attractive models to study this syndrome, as they, too, develop UV radiation-induced skin tumors, mimicking the human phenotype. Recombinant adenovirus carrying the human XPA gene was used for in vivo gene therapy in UVB-irradiated skin of such mice. Virus s.c. injection led to the expression of the XPA protein in basal keratinocytes and prevented deleterious effects in the skin, including late development of squamous cell carcinoma. Thus, efficient adenovirus gene delivery to the skin is a promising tool for reconstitution of specific DNA repair defects in XP patients.

Original languageEnglish (US)
Pages (from-to)17759-17764
Number of pages6
JournalProceedings of the National Academy of Sciences of the United States of America
Issue number51
StatePublished - Dec 21 2004


  • DNA repair
  • Gene therapy
  • Keratinocytes
  • Ultraviolet

ASJC Scopus subject areas

  • General


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