Word: ada
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...latest experiment at the NIH, Blaese administered a drug to one of his two original Ohio patients that coaxed some stem cells out of the bone marrow and into her bloodstream. Extracting blood, he painstakingly separated out the rare stem cells, inserted normal ADA genes into their DNA and injected the cells back into the girl's bloodstream, hoping that they would migrate back to the marrow and take up permanent residence...
Although Richard and Lori Riggins, of Exeter, California, have a normal four-year-old daughter, a son born to them in 1991 was diagnosed with SCID at the age of four weeks, and ever since has required treatment with PEG-ADA to survive. His disorder was evidence that both his mother and father, while healthy themselves, carried a recessive gene for SCID. This meant that any of their offspring would have a 1-in-4 chance of being stricken with the disease. The outlook was equally gloomy for Crystal Emery and Leonard Gobea, from California's Imperial Valley; their first...
Aware of the risk, both mothers chose to have amniocentesis after becoming pregnant again last fall. The test results showed that neither fetus was producing ADA and that the babies would have SCID. It was then that Dr. Diane Wara, the pediatric immunologist who had treated the Rigginses' other child, suggested the stem-cell trial. Lori Riggins was easily convinced. "You only get a once-in-a-lifetime chance to get large amounts of stem cells," she says. "That's at birth, and we didn't want to pass up that chance...
...part of the experiments. Immediately after Andrew was born, the obstetrician snipped his cord and drew out the umbilical blood. She rushed it to Childrens Hospital in Los Angeles, where a team led by Drs. Donald Kohn and Kenneth Weinberg separated the stem cells and endowed them with normal ADA genes. Then the newly equipped stem cells were injected into the baby's bloodstream. Two days later, Wara went through the procedure on Zachary Riggins in San Francisco, after his stem cells had been shuttled to Kohn and Weinberg in Los Angeles for genetic engineering...
...guard against the possibility that the gene therapy will not work, doctors will initially treat both infants with weekly injections of PEG-ADA. "We have no intention of letting these children get sick while we're waiting to see if the stem cells ((become functional))," says Wara. "When we see that this has happened, then we will start withdrawing the enzyme replacement." But will it happen? "My personal hunch is that this is going to benefit these two children," says Kohn. "If it does, then we can go on to more common diseases...