Gene therapy progress and prospects: cancer gene therapy using tumour suppressor genes

Gene Ther. 2004 Mar;11(6):497-503. doi: 10.1038/


Targeting tumour suppressor gene pathways is an attractive therapeutic strategy in cancer. Since the first clinical trial took place in 1996, at least 20 other trials have investigated the possibility of restoring p53 function, either alone or in combination with chemotherapy, but with limited success. Other recent clinical trials have sought to harness abnormalities in the p53 pathway to permit tumour-selective replication of adenoviral vectors such as dl1520 (Onyx-015). Other tumour suppressor genes, such as retinoblastoma (Rb) and PTEN (phosphatase, tensin homologue, deleted on chromosome 10), are the targets for imminent clinical trials, while microarray technologies are revealing multiple new genes that are potential targets for future gene therapy.

Publication types

  • Review

MeSH terms

  • Gene Targeting
  • Gene Transfer Techniques
  • Genes, Tumor Suppressor*
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage
  • Humans
  • Neoplasms / therapy*