title: Potential of Advexin: a p53 gene-replacement therapy in Li-Fraumeni syndrome.
abstract: Li-Fraumeni syndrome is a rare autosomal dominant cancer predisposition syndrome. The majority of families fulfilling definition of Li-Fraumeni syndrome demonstrate inherited abnormalities involving the p53 gene. Cells with dysfunctional p53 are predisposed to the development of cancer phenotype. Advexin (Introgen Therapeutics Inc., TX, USA) is an adenoviral-based experimental therapeutic that provides delivery of wild-type p53 to cancer cells and demonstrates anticancer activity following adequate expression of p53. Theoretically, correction of p53 function in cancer developing in patients with Li-Fraumeni syndrome through treatment with Advexin will provide anti-tumor activity. One patient with Li-Fraumeni syndrome has been reported to have responded to Advexin. This review will summarize background knowledge of Li-Fraumeni syndrome, mechanisms of Advexin and clinical response of cancer to Advexin with a focus on Li-Fraumeni syndrome.
authors.affiliation: Mary Crowley Cancer Research Centers, 1700 Pacific Avenue, Ste 110, Dallas, TX 75201, USA. jackie.nemunaitis@gmail.com
authors.forename: Jackie M
authors.initials: JM
authors.lastname: Nemunaitis
authors.forename: John
authors.initials: J
authors.lastname: Nemunaitis
mesh_terms: D000328:Adult; D005260:Female; D020022:Genetic Predisposition to Disease; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D016864:Li-Fraumeni Syndrome / Q000235:genetics / Q000628:therapy*; D016159:Tumor Suppressor Protein p53 / Q000235:genetics*; D014765:Viral Vaccines / Q000627:therapeutic use*
chemical_list: D016159:Tumor Suppressor Protein p53; D014765:Viral Vaccines; C512972:advexin