Diagnosing and Treating Genetic Diseases
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Drugs, vaccines, and enzyme replacements created by genetic research are already widely used to treat people for diseases. "Recombinant DNA" technology decreases health care costs by providing large amounts of widely used drugs, like insulin and human growth hormone. In this section, learn how recombinant DNA works and about some of the therapies developed from genetic research.

What is Recombinant DNA?

Recombinant DNA is DNA that has been altered in the laboratory so that a new gene has been added or an existing gene deleted. This process is usually conducted on a bacterium (one-celled organisms). A gene is inserted into the bacterium that contains the instructions for the bacterium to produce a protein that it would not have produced naturally. The bacterium's DNA is further altered so that it will not survive outside the laboratory. By using recombinant DNA, large quantities of proteins such as insulin for diabetics can be mass produced. Bacteria become protein-making factories.


rTPA: A Recombinant DNA Clot-Buster Drug
Blood clots (clumping of the blood cells so that they cannot flow through the veins and arteries) cause heart attacks, strokes, lung embolisms, and deep vein thrombosis. Doctors used a drug called streptokinase to dissolve the clots but had problems with how streptokinase works. Streptokinase affects blood vessels in the entire body, which can increase bleeding during emergency operations. Doctors wanted a drug that would affect only a small area at a time.

 
Heart patient operation.
Courtesy of the National Library of Medicine
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Revolution in Progress: Human Genetics and Medical Research/
National Institutes of Health