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Areas of Biotechnology Research

Health Care

In addition to the use of recombinant DNA to make bacteria produce medicines, biotechnology can improve human health in other ways. A mutation is a change in an organism's DNA. Sometimes, mutations lead to characteristics that are good for the organism. Other times, mutations can cause disease. Gene therapy is a kind of biotechnology that introduces healthy genes into humans to counteract malfunctioning, disease-causing genes.

Designing a gene therapy is a multistep process. First, an appropriate gene for the treatment must be identified. Researchers must then create and purify large quantities of it. Next, they must figure out how to get it into living cells. The treatment must be tested on cells grown outside organisms, then on animals, and finally on humans.

Gene therapy does not replace the unhealthy gene. It supplements it with a healthy gene that can make useful proteins. Researchers hope that the healthy gene will be able to reduce the effect of the unhealthy gene.

Tests of some gene therapies have caused illness or death among some humans who have volunteered for medical trials. China is the only country that has approved a gene therapy, targeted at head and neck cancers, for widespread use. But many companies are currently designing and testing gene therapies for humans. Gene therapy has also been used to successfully treat genetic blindness and blood clotting disorders in dogs.

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