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A Third Revolution in Modern Medicine, Part Two
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12121 |
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Section : |
NATURAL SCIENCE
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| Issue
Date : |
12 / 1987 |
4,147 Words |
| Author
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Wolfgang Sadee
Wolfgang Sadee is professor of pharmacy and pharmaceutical
chemistry at the University of California, San Francisco and
editor of Pharmaceutical Research. |
Editor's note: In the November 1987 issue, Part One of this two-part series provided background on the knowledge and technologies of the protein domain.
"Bubble Boy Dies" read the headlines a few years ago. The popular press had been filled with references to a boy whose body had no defenses to protect it from foreign substances, so he had to live in strict isolation. Yet in the end, despite all precautions, he succumbed to a simple infection that somehow penetrated his protective environment.
The boy was suffering from a genetic disorder in which his body failed to produce one particular enzyme (a type of protein) essential for the function of his immune system. Today, medical researchers are rapidly approaching a level of expertise that will permit them to provide that needed enzyme or even to change the boy's genetic structure so that his own body would produce the enzyme.
Recent developments in biology have revealed much of the structure and function of DNA and of proteins. DNA is the carrier of genetic information, and proteins are the building blocks essential to life that are encoded by the DNA contained in some 100,000 genes for each human. (Part I explains these concepts in detail.) We are now beginning to experience the impact of these new insights on all medical fields, with one area of great promise being the direct application of the body's proteins as novel drugs.
One well-known protein used as a drug is the hormone insulin, prescribed for treating diabetes. However, until 1982 insulin had to be extracted at high cost from large numbers of animal pancreases. Now with recombinant DNA technology, it is possible to introduce the human insulin gene into bacteria for mass production of this much-needed hormone. Indeed, after it was shown that the recombinant insulin is no different from native insulin, insulin became the first recombinant---DNA-produced protein (or peptide) drug to be approved for marketing by the Food and Drug Administration (FDA). However, because of the complexity of proteins, and the uncertainties associated with their use as potential drugs, there are presently only four recombinant proteins that have been approved by the FDA.
White insulin was already available for the treatment of diabetes before the development of recombinant insulin, recombinant technology has made it possible to produce large quantities of pure human insulin, thus avoiding the defensive reactions that are sometimes
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