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Genes to the Rescue
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10335 |
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Section : |
NATURAL SCIENCE
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| Issue
Date : |
12 / 1993 |
3,261 Words |
| Author
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Ricki Lewis
Ricki Lewis is the author of two college textbooks, Human
Genetics: Concepts and Applications and Life (1997, 1998,
McGraw-Hill College Publishing, Dubuque, Iowa), and coauthor
of two anatomy and physiology textbooks. She has published
more than 2,500 articles in popular magazines and journals. |
Just a few years ago, the idea of replacing defective genes--gene therapy--seemed futuristic, the stuff of science fiction. The first human clinical trials of gene therapy began in 1990. Today, scientists regard gene therapy not as some exotic, far-off option for when traditional measures fail hut as the very future of medicine. Robert Hemley, a geneticist at Delphi Group, a biotechnology consulting firm in White Plains, New York, says he is astonished at how rapidly gene therapy protocols on paper have become reality, echoing the thoughts of many researchers.
"Will gene therapy become part of standard health care? Absolutely!" says Mark Pearson, president of Darwin Molecular Corporation, a new biotechnology company in Kirkland, Washington, planning to use information from gene discoveries to develop new drug treatments. "There will be fantastic advances in medicine as we start to define the molecular basis of many diseases," he adds.
Gene therapy research is proceeding rapidly with a plethora of alternative gene-delivery options being explored. These include several different types of viruses, minute spherical projectiles, microscopic fat globules, and jolts of electricity. A look at the development of one of these options illustrates the challenges and promise of gene therapy.
A harmless virus
In 1991, Arun Srivastava, an associate professor of microbiology and immunology at the Indiana University School of Medicine in Indianapolis, realized that adeno-associated virus (AAV), the very "safe" virus he was studying, might be a good vehicle (vector) for delivering a healthy gene to replace a faulty one--the essence of gene therapy. Unlike most viruses, which usurp the cell's protein-synthesizing machinery to make more of themselves, eventually bursting the cell and releasing many viruses, AAV seems content to spread quietly, taking up residence in human cells of all kinds without causing any known ill effects. Also, in 1991, researchers Ken Berns of Cornell Medical School and Richard Samulski, then at the University of Pittsburgh, determined that AAV nestles into a certain spot on chromosome 19. (The 23 human chromosomes are numbered from largest to smallest.) "AAV is defective. It doesn't do any damage," Srivastava says. So, because the virus harmlessly inserts into a human chromosome, why not hook a gene to it and send the duo into a cell needing the gene?
Sickle-cell disease came to mind, because its underlying defect has been
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