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Boys with rare, fatal brain disease may benefit from stem cell gene therapy

Researchers found that 15 of 17 boys with adrenoleukodystrophy, a rare and fatal neurological illness that affects males with just one copy of the X chromosome, who underwent stem cell gene therapy had normal functioning and no major disability after two years. The findings in The New England Journal of Medicine suggest that stem cell gene therapy is as effective as and possibly safer than stem cell transplants, according to an accompanying editorial.

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