Word: ashanthi
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...outward appearances, Ashanthi ("Ashi") DeSilva is a normal, healthy 12-year-old who loves sports and would rather play basketball than do her seventh-grade homework. But Ashi holds a unique place in medical history: she is the first recipient of successful gene therapy...
...gene therapy, most of the current "smart bombs," as in the case of Ashanthi DeSilva, are viruses, which by their nature invade cells and deposit their genetic material into the cell nucleus. Researchers have learned how to strip the viruses of their reproductive genes, insert into the viral DNA the beneficial gene they want to deliver, and then let the virus infect a patient's cells. The virus inserts its own now harmless genes, as well as the beneficial one, into the cellular DNA. If all goes well and the gene "expresses" itself, the cell begins producing the needed protein...
...Even when the rare stem cells are found, inserting genes into them is difficult because they divide infrequently, and the vectors used in gene therapy insert genes only into cells that are dividing. Had Anderson's group been able to use stem cells in the landmark therapy with little Ashanthi DeSilva, for example, follow-up treatments would not have been necessary. Endowed with the normal gene, the marrow stem cells would have produced a continuing supply of new white blood cells carrying that gene, and Ashanthi's cure would have been permanent...
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