https://www.reddit.com/r/science/comments/1knfkio/worlds_first_personalized_crispr_therapy_given_to/
KJ Muldoon was born with severe CPS1 deficiency, a rare disorder that stops the body clearing ammonia. A team at Children’s Hospital of Philadelphia and Penn Medicine built a base editor, a precise CRISPR variant, designed to fix his specific mutation. It was delivered to his liver in lipid nanoparticles and made in about six months.
He got his first dose in February 2025 and follow-ups in March and April. Results were published in the New England Journal of Medicine on May 15. So far he can eat more protein, needs less medication, and has had no serious side effects. Doctors stress it’s early and he will need long-term follow-up.
The bigger win may be the template: a fast route to one-off gene edits for single patients.
Source: www.reddit.com · Also: NEJM paper · Penn Medicine · NBC News (photo)