ABSTRACT

Cancer has become a focus of efforts in genetic therapy. While a genetic basis for cancer has been suspected for many years, the discovery of the retinoblastoma gene solidified the role of defective genes in cancer. The most straightforward approach to genetic therapy is to replace the DNA segment corresponding to a gene that is missing or damaged. An ingenious application of gene transfer technology was the introduction of genes into cancer cells that make them more sensitive than normal cells to a specific treatment. The future of treatment for cancer in general and for ocular tumors in particular is likely to involve genetic therapy. Gene replacement therapy, gene inhibition therapy, suicide gene therapy, viral oncolytic therapy, and targeted small-molecule therapy comprise a group of therapeutic approaches that have in common a rationale based on detailed molecular understanding of the cancer of interest.