How a custom-made gene therapy could save one baby's life

Baby KJ Muldoon in hospital in April 2025 Credit: Chloe Dawson

A baby born with a rare, life-threatening genetic disease is now thriving after receiving an experimental gene-editing treatment.

Baby KJ Muldoon was born with CPS1 deficiency, a condition that affects around one in a million babies.

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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