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Gene Therapy May Reverse Hurler Syndrome, a Rare and Severe Illness in Kids
Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
In a study of eight children with the condition, called Hurler syndrome, researchers found that the gene therapy was safe over two years. It also showed potential for beating the current standard treatment, stem cell transplantation.
If the gene therapy proves safe and effective in the longer term, experts said, it may well replace stem cell transplants in the future.
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