CHAPEL HILL -- Even if the patients hadnt been as young as 4 months old, the surgery would have been harrowing: six holes bored into the skull, six tiny tubes inserted directly into targeted parts of the brain, then a solution containing hundreds of millions of viruses pumped in. But the rare degenerative illness it fights is even scarier. Canavan disease strikes infants, essentially making the brain attack itself with a toxic chemical, stopping and reversing development. It then kills, usually before age 10. The procedure used in the study though, slows Canavans progress and improves and may even help extend their lives, according to a study that appeared last month in the online journal Science Translational Medicine. It does that by using viruses as microscopic trucks to deliver missing genetic material precisely where its needed in the kids brains. This form of gene therapy was created at UNC-Chapel Hill, and the viral vehicle and genetic cargo used in the study were developed there at the medical schools Gene Therapy Center. Center director R. Jude Samulski was a senior author of the study, which began in 2001 and tracked 13 children who received the treatment. The youngest was 4 months … Continue reading
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