Gene Therapy for Cancer

Cancer occurrs by the production of multiple mutations in a single cell that causes it to proliferate out of control. Cancer cells often different from their normal neighbors by a host of specific phenotypic changes, such as rapid division rate, invasion of new cellular territories, high metabolic rate, and altered shape. Some of those mutations may be transmitted from the parents through the germ line. Others arise de novo in the somatic cell lineage of a particular cell. Cancer-promoting mutations can be identified in a variety of ways. They can be cloned and studied to learn how they can be controlled.

Several methods such as surgery, radiation, and chemotherapy have been used to treat cancers. The cancer patients who are not helped by these therapies may be treated by gene therapy. Gene therapy is the insertion of a functional gene into the cells of a patient to correct an inborn error of metabolism, to alter or repair an acquired genetic abnormality, and to provide a new function to a cell.

Two basic types of gene therapy have been applied to humans, germinal and somatic (1). Germinal gene therapy, which introduces transgenic cells into the germ line as well as into the somatic cell population, not only achieve a cure for the individual treated, but some gametes could also carry the corrected genotype. Somatic gene therapy focuses only on the body, or soma, attempting to effect a reversal of the disease phenotype by treating some somatic tissues in the affected individual.

One of the most promising approaches to emerge from the improved understanding of cancer at the molecular level is the possibility of using gene therapy to selectively target and destroy tumor cells, for example, the loss of tumor suppressor genes ...

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