Word: geneses
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The initial goals of gene therapists were to cure relatively straightforward genetic disorders, such as Huntington's disease and sickle-cell anemia, that are 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...
But how do you get a new gene into the nucleus of a cell? The trick, researchers discovered early on, is to take advantage of the infectious power of viruses; burrowing into cells is second nature to them. A virus is nothing more than a tiny strip of DNA or...
"You can do spectacular things with cells in a laboratory dish," explains Anderson. "You can easily get the genes in, change the cell's properties and do other things that ought to enable you to treat disease successfully." That is precisely what Anderson and his colleagues did eight years ago...
In an early gene-therapy trial for cystic fibrosis, inflammation caused by the viral carrier, an altered adenovirus, was so severe that the FDA ordered a halt to the effort, casting a pall over all the other trials--and the field in general. More problems plagued the researchers. In many...
Most researchers in the field agree that the adenovirus and retrovirus vectors are imperfect, to say the least. In addition to having immunological side effects, both lack the carrying capacity to accommodate the larger, more complex genes that would be useful in therapy. "There are only three problems in gene...