Gene Therapy

Perhaps the ultimate application of recombinant DNA technology is gene therapy, the direct transfer of genes into humans to treat disease. When the first recombinant DNA experiments with bacteria were announced, many researchers recognized the potential for using this new technology in the treatment of patients with genetic diseases. But, before recombinant DNA could be used on humans, a number of difficult obstacles had to be overcome. The genes responsible for particular genetic diseases needed to be located and cloned, and special vectors had to be developed that would reliably and efficiently deliver genes to human cells.

In 1990, gene therapy became reality. W. French Anderson and his colleagues at the U.S. National Institutes of Health (NIH) transferred a functional gene for adenosine deaminase to a young girl with severe combined immunodeficiency disease, an autosomal recessive condition that produces impaired immune function.

Today, thousands of patients have received gene therapy, and many clinical trials are underway. Gene therapy is being used to treat genetic diseases, cancer, heart disease, and even some infectious diseases such as AIDS. All of these

[Table 18.6 Vectors used in gene therapy





Efficient transfer

Transfers DNA only to dividing

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