Nature
Genomics
Gene therapy saved a boy's life — but then something unexpected happened in his brain
Like when a firefighter puts out one fire but accidentally knocks over a lamp: the treatment worked brilliantly at what it was designed to do, but researchers are now asking whether something else went wrong.
This means scientists need to watch more carefully for subtle side effects in gene therapy, especially in the brain, as we push these powerful treatments into the clinic.
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