Pending investigation, the FDA has halted two similar gene experiments. The tone changed on the second day, however, with bureaucratic language and accusations on both sides giving way to pleas from doctors and patients alike for a continuation of human gene-therapy experiments. I hate being sick. The Promise of Cures Humans are a composite of approximately 50,000-100,000 genes, which determine physical characteristics as well as vulnerability to disease. The NIH and industry are racing to locate and understand the role of every single human gene, an endeavor known as the Human Genome Project.
Gene therapy works by replacing a person's defective genes with healthy genes.
Researchers have discovered the genes associated with many diseases, including ones for Alzheimer's disease and a hearing disorder called Waardenburg's syndrome. Neither has a cure. Packing Genes Into a Virus A common way to inject healthy genes into the body, Patterson said, is to pack them into a weakened cold virus called an adenovirus. This cold virus will invade the body, as it always does, and eventually be killed by the body's own natural defense system. But before the cold virus dies, it transports the healthy gene into a target cell.