Gene therapy might promise an end to the fatal "Bubble Boy" disease, possibly curing 8 out of 10 children suffering from the disorder. The 8 kids who received treatment for the rare but deadly condition were able to discontinue their expensive and sometimes ineffective medications during the 4 year follow-up period.
Bubble Boy disease, which affects up to 100 babies born in the U.S. alone, is the result of severe immune system deficiency; most babies affected don't survive past childhood. One of the most famous cases was that of Houston's David Vetter, who lived most of his 12 years behind plastic to cut down on his exposure to germs.
Gene therapy, from the studies done in Italy and Israel, was used on patients under the age of 2. The "researchers removed marrow cells from the patients, equipped the cells with working copies of the gene for the enzyme, and injected the cells back into the patients." (Malcolm Ritter, AP Science, Wednesday January 28)
There are 2 seperate forms of the disorder, and gene therapy seems to show promising results for both, but with a leukemia risk as a side effect for one form. None of the kids in the study showed any signs of leukemia or any other negative side effects.
The study is important because it shows promise as a possible cure not only for this rare disease, but for more common conditions such as sickle cell disease and related blood conditions.
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