Archive · 19 May 2025
Baby Treated with Personalized Gene Editing for Rare Condition
A baby boy in the US, named KJ Muldoon, has received a special treatment for a rare illness.
He has a condition that prevents his body from getting rid of waste, like a clogged drain.
Doctors used a new method, like molecular scissors, to fix a mistake in his genes.
These "scissors" are part of a technique that won a Nobel Prize.
The treatment was made just for him.
It's helping him get better and he can now eat foods that were previously off-limits.
Doctors hope this new method will help many more kids in the future.
A US infant with CPS1 deficiency has been treated with a personalized gene-editing technique.
The treatment uses CRISPR-Cas9, a technique that won the Nobel Prize in 2020.
The infant's condition prevented his body from eliminating toxic waste produced by his metabolism.
The tailored infusion targets the boy's specific genetic mutation.
The treatment allows the baby to consume a diet richer in proteins and reduces the need for medication.
- Who
- KJ Muldoon, a baby with CPS1 deficiency, and his medical team.
- What
- Personalized gene-editing using CRISPR-Cas9 to treat CPS1 deficiency.
- When
- Treated in 2025, study published May 15, 2025.
- Where
- Children's Hospital of Philadelphia, USA.
- Why
- To correct a genetic mutation causing CPS1 deficiency.
- Patient
- KJ Muldoon, a 9.5-month-old boy
- Condition
- CPS1 deficiency
- Treatment
- Personalized gene-editing using CRISPR-Cas9
- Treatment Method
- Infusion of molecular scissors to fix the gene mutation in the liver
- Date of Study Publication
- May 15, 2025