Word: retroviruses
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...effect a cure, doctors would remove bone-marrow cells from a patient and expose them to a retrovirus* engineered to carry correctly functioning versions of the patient's faulty gene. When the retrovirus invaded a marrow cell, it would insert itself into the cellular DNA, as retroviruses are wont to do, carrying the good gene with it. Reimplanted in the marrow, the altered marrow cells would take hold and multiply, churning out the previously lacking protein and curing the thalassemia patient...
...gene be slipped into the middle of another vital gene, for example, it might disrupt the functioning of that gene, with disastrous consequences. Also, says M.I.T. biologist Richard Mulligan, there are limitations to the viral insertion of genes. "Most genes," he explains, "are too big to fit into a retrovirus...
...Central Africa and New Guinea, it has been linked to Burkitt's lymphoma, an immune-cell cancer that primarily strikes children. In southern China, the virus plays a role in nasopharyngeal carcinoma, a malignancy of the nose and throat that afflicts more than 50,000 people a year. Retroviruses are known to cause cancer in a wide range of animals, from mice to chickens. In the early 1980s, Dr. Robert Gallo and his co-workers, as well as a group of Japanese researchers, showed for the first time that a retrovirus is responsible for cancer in humans. The culprit...
Although experimental work on mammals is proceeding slowly, Richard Mulligan, who has been practicing gene therapy on mice at the Whitehead Institute, is optimistic. "We are pretty damn close," he says. "We have retrovirus vectors that transfer efficiently. It looks like we can infect the ( appropriate types of cells reasonably safely." But, he concedes, he has not yet been able to induce the genes to "turn on" and order the cells to produce the missing proteins...
...solution, Mulligan hopes, is to attach the protein's gene to a section of DNA that acts as an on-switch, or promoter, before implanting it in the retrovirus. In his experiments to date, he says, the strategy is "working fantastically," and he expects encouraging results within two or three months...