Video Doctors Save Baby's Life with First-Ever Gene Fix for Deadly Rare Disease

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.

Doctors edited a baby's DNA to treat a serious hereditary disease.

Video Doctors save baby's life with first-ever gene fix for deadly rare disease

The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific gene mutation, doctors say.

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Many rare hereditary diseases have so far been considered incurable, but the successful treatment of a baby in Philadelphia shows that this could soon change.

Tailored gene therapy has been used for the first time on a young child, with a team of American doctors announcing success in a therapy that was completely unimaginable just a few years ago.