Word: geneses
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How 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...
Some researchers look forward to the day when gene therapy is used to repair damaged genes. With the new vectors, they would infect cells with small molecules that combine DNA and RNA. These hybrid molecules would seek out and bind to the defective gene, enabling it to function normally. "It...
The other new vector is being fashioned by Salk's Verma. "What we want," he says, "is a virus that is easy to make, that delivers genes at very high efficiency, that can infect a nondividing cell and that enables its therapeutic gene to become part and parcel of the...
Seeking the best candidate, Verma zeroed in on the most notorious of the retroviruses--HIV, the virus that causes AIDS. He eliminated the protein envelope that allows the virus entry into T cells, substituted one enabling it to infect a greater variety of cells, and removed the six genes that...
Gene therapists are looking even further ahead. Pennsylvania's Wilson predicts that the next advance will be a mechanism built into the vector to regulate the expression of a therapeutic gene, turning it on or off. "Most diseases and most drugs require modifying the dose," he explains, "but the genes...