US EditionCRISPR Fulfills Its Promise with First-Ever Personalized Gene-Editing Therapy
- Doctors at the Children’s Hospital of Philadelphia treated 9-month-old KJ Muldoon in February 2025 using personalized CRISPR gene-editing for CPS1 deficiency, a rare genetic disorder.
- KJ faced a fatal outlook due to CPS1 deficiency disrupting ammonia removal, and his doctors sought a gene-editing solution after liver transplant was the only prior option.
- The therapy involved three custom-designed CRISPR infusions targeting KJ’s unique mutation, delivering molecular scissors to his liver to correct the genetic error.
- After treatment, KJ can eat more protein, needs fewer medications, and shows significant improvement, with his mother reporting that he is gaining weight and thriving.
- This first-ever personalized CRISPR treatment marks a milestone in gene therapy, suggesting scalable potential for treating rare diseases, though KJ requires lifelong monitoring.
15 Articles
15 Articles
US Baby Becomes First to Undergo Successful Personalized Gene Therapy
A newborn baby. Credit: Melimama / CC BY 2.0 An American baby, KJ Muldoon, born with a rare and life-threatening genetic disease, is growing normally after undergoing the first successful personalized gene therapy. In a new study, researchers report that the baby is among the first humans to receive this therapy to correct tiny yet critical errors in his genetic code. These errors are fatal to half of the affected infants. Although this treatmen…
Breakthrough gene editing treatment helps child born with rare disorder
Doctors announced this week that they have treated a newborn baby with a rare genetic disease using the world’s first personalized gene editing therapy. Geoff Bennett discussed the treatment and its potential with Dr. Peter Marks. He oversaw gene therapy treatment and vaccine safety and approval for the FDA before he left in March.
Breakthrough gene-editing treatment saves baby
What happened A team of doctors and scientists has used a tailor-made gene-editing therapy to treat an infant with a rare genetic condition, a medical first that opens the door to a new era of individualized medicine, especially for people with uncommon diseases, the researchers reported Thursday in The New England Journal of Medicine. "This is the future of medicine," said study coauthor Dr. Kiran Musunuru, a gene-editing expert at the Universi…
In a World First, CRISPR Drug Tailored for One Baby Shows Life-Saving Promise
Scientists used CRISPR tech to create a custom-made drug targeted at helping a newborn baby fight a rare genetic disorder. According to an extraordinary new paper published in The New England Journal of Medicine, the drug worked — marking the first time that a patient was successfully healed with a bespoke gene-hacked medicine, and a possible watershed moment for the field of genetic medicine. The baby, named KJ Muldoon, was born at the Children…
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