SPECIAL FEATURE
February 2025 Volume 24 Issue 1
Baby saved by world’s first personalized gene therapy
ShareDoctors have saved a baby’s life using a gene therapy created specifically for one individual patient — opening the door to a new era of personalized medicine. Baby KJ Muldoon was born with a rare genetic condition that caused dangerous levels of ammonia to build up in his blood, putting him at high risk of brain damage or death.
Standard treatments offered only temporary control, and he was too small for a liver transplant. Doctors at the Children’s Hospital of Philadelphia turned to an experimental approach based on CRISPR gene editing. Using a “molecular pencil,” they corrected a single faulty DNA letter in KJ’s gene — effectively fixing the root cause of his disease. The therapy was custom-designed for KJ’s exact mutation, making this the first time a personalised gene-editing treatment has been used in a patient.
Following treatment, KJ’s condition stabilized, and he is now meeting developmental milestones. Experts say this breakthrough could eventually help people with ultra-rare genetic disorders that currently have no cure. While not all genetic diseases will be treatable this way, researchers believe personalized gene therapies may transform care for many families in the years ahead.
[Source: Science News, Children’s Hospital of Philadelphia]
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