USAResearch and clinical trials
Gene-editing therapy custom-designed for a single baby used for the first time
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In the USA, a baby named KJ with severe CPS1 deficiency received an individualized base-editing therapy. The treatment was given by a team from Children's Hospital of Philadelphia and Penn Medicine. It was designed solely to correct the baby's own gene change. It was prepared in about six months. Three doses were given between February and April 2025. The baby was reported to tolerate more protein. The need for medication was also reported to have decreased. The results were published in NEJM.
What does it mean for patients and families?
This is an example of a method tailored to a single patient. Similar individualized treatments are not yet routine. However, clinical studies are being planned.
Source
Scientific paper: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. New England Journal of Medicine, 2025. DOI: 10.1056/NEJMoa2504747 · PubMed 40373211
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: CPS1 deficiency (urea cycle disorder)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.