A baby in Pennsylvania with a rare, life-threatening genetic disorder is showing remarkable progress after receiving a personalized gene editing treatment.
This groundbreaking therapy targets a tiny error in his DNA that typically proves fatal for half of affected infants.
KJ Muldoon was born with CPS1 deficiency, a condition affecting about one in a million newborns.
The disorder prevents the body from removing toxic ammonia from the bloodstream, often requiring a liver transplant or leading to early death.
Using an advanced gene editing technique called base editing, doctors at Children’s Hospital of Philadelphia created a custom therapy to correct KJ’s faulty gene.
Unlike earlier CRISPR methods that cut DNA, base editing precisely flips a single DNA “letter,” reducing risks of unintended changes.
Since receiving the treatment via IV infusions beginning in February, KJ, now nine months old, has shown encouraging signs. He eats more normally, handles common illnesses better, and requires less medication.
His parents celebrate every developmental milestone, knowing how precarious his early prognosis was.
While researchers caution that long-term effects remain under observation, this case marks a hopeful advance for treating rare genetic diseases with personalized gene therapy. It also suggests such treatments may become more accessible as production improves and costs decrease, potentially benefiting millions worldwide with rare conditions.
Related topics:
- Smartphone Overuse Linked To Lower Happiness In Kids & Teens
- Nearly 1 In 4 U.S. Kids Live With Parents Struggling With Substance Use Disorder
- At What Stage Is a Cold Contagious for a Kid?
