Word: heart
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Dates: during 1990-1999
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...caused by a single defective gene. The strategy was simple: substitute a normal gene for a faulty one. But scientists quickly realized that adding genes to cells could also impart new functions to those cells. That may lead to the genetic treatment of a host of other disorders, including heart disease and many forms of cancer...
...heart-patient trial, St. Elizabeth's Isner found a novel way around the delivery problem. Eschewing virus carriers, he fashioned a construct called "naked DNA." It consists of part of a human gene called VEG-F, which stimulates the growth of blood vessels, and includes its signal segments. These segments, Isner explains, "order the cell, once it has manufactured the gene product, to export it from the cell...
...Phase I trial, Isner injected a saline solution containing his naked DNA through a small "keyhole" incision in the chest of his heart patients and directly into their heart muscle. A few weeks later, tests on everyone in the trial group showed greatly improved blood flow to the heart muscle though tiny new blood vessels that bypassed clogged arteries...
...does the naked DNA, without viral assistance, penetrate the walls of the heart-muscle cells? "To be perfectly honest," Isner confesses, "no one really understands how it gets there." But unlike most other therapeutic genes, which must find their way into millions of cells to have a therapeutic effect, VEG-F needs to invade only relatively few. Its protein product, issuing from the cell, can act on untold numbers of surrounding, untreated cells. Quips Isner in a parody of the Marine Corps slogan, "All we're looking for are a few good cells...
...this untapped potential could be a terrific boon to medicine. Most diseases involve the death of healthy cells--brain cells in Alzheimer's, cardiac cells in heart disease, pancreatic cells in diabetes, to name a few. If doctors could isolate stem cells, then direct their growth, they might be able to furnish patients with healthy replacement tissue...