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How a custom-made gene therapy could save one baby's life
A baby born with a rare, life-threatening genetic disease is now thriving after receiving an experimental gene-editing treatment. He's one of the first to be successfully treated with a bespoke therapy targeting a tiny but deadly error in his DNA. His doctors hope the technology could one day help the estimated 350 million people worldwide with rare diseases.
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By SBSA baby born with a rare, life-threatening genetic disease is now thriving after receiving an experimental gene-editing treatment. He's one of the first to be successfully treated with a bespoke therapy targeting a tiny but deadly error in his DNA. His doctors hope the technology could one day help the estimated 350 million people worldwide with rare diseases.