Molecular genetics of childhood cancer: Implications for pathogenesis, diagnosis, and treatment

Molecular characterization of translocation break-points has identified an array of oncogenes and tumor suppressor genes in childhood cancers, leading to new and often profound insights into the mechanisms of malignant transformation. The cloning of these genes has also contributed to the development of molecular diagnostic assays that have greatly improved clinical treatment capabilities, including the detection of minimal residual disease. Many current studies focus on oncogenic transcription factors and their downstream targets, which may prove to be molecules susceptible to novel, tumor-specific therapies. Ultimately, it may be possible to replace inactivated tumor suppressors with fully functional genes or to induce leukemic cells to undergo normal differentiation and apoptosis. Results in genetically modified mice suggest that such manipulations are attainable future goals.