Baby gets custom CRISPR gene therapy for rare liver disorder
A seven-month-old baby named KJ just became the first person to get a custom CRISPR gene therapy for a rare liver disorder called CPS1 deficiency.
This condition causes dangerous ammonia buildup that can lead to brain damage or death.
Doctors at Children's Hospital of Philadelphia developed and delivered the treatment in just six months.
Therapy fixed the mutation behind KJ's illness
KJ's therapy used lipid nanoparticles to deliver gene-editing tools directly to his liver cells, fixing the mutation behind his illness.
After treatment, KJ could safely eat more protein and needed fewer meds to control ammonia.
He also bounced back from infections that are usually very risky for kids with this disorder.
Breakthrough hints at a future for rare disease treatments
KJ got three doses with no serious side effects so far, showing what's possible when gene therapy is tailored quickly for rare diseases.
His progress is being closely watched, but this breakthrough hints at a future where fast, personalized treatments could help others facing similar genetic challenges.