Scientists Expand Personalized Gene-Editing Therapy That Saved Baby KJ Muldoon
Researchers at Children's Hospital of Philadelphia and Penn Medicine are preparing a clinical trial to give at least five more children a personalized CRISPR base-editing therapy modeled on the one that saved infant KJ Muldoon, who was born with a rare metabolic disorder called carbamoyl phosphate synthetase 1 (CPS1) deficiency that carries roughly a 50 percent infant mortality rate. Doctors designed and manufactured KJ's bespoke treatment in about six months in early 2025, correcting his specific gene mutation in liver cells using base editing, an offshoot of CRISPR that makes single-letter DNA changes without cutting the strand. Cardiologist Kiran Musunuru, one of KJ's doctors, has said the team believes it can shorten that turnaround even further as the approach scales to more patients.
The push toward one-patient, custom-built gene therapies is broadening beyond Philadelphia. A new Center for Pediatric CRISPR Cures launched this year at the University of California, Berkeley and the University of California, San Francisco with the same goal of building bespoke editing therapies for individual children with ultra-rare mutations. In September, the federal Advanced Research Projects Agency for Health, known as ARPA-H, announced two programs to fund research into manufacturing this kind of "precision genetic medicine" at scale.
What supporters say:
Scientists like Jennifer Doudna, the CRISPR pioneer and Nobel laureate, have called the approach a new model for treating previously untreatable ultra-rare genetic diseases on a patient-by-patient basis.
Backers point to KJ Muldoon's six-month turnaround as proof the process can move fast enough to help infants before their conditions become fatal, and researchers say future cases could move even quicker.
What critics say:
Each therapy is built from scratch for a single patient, and it remains unclear how insurers or health systems would price or scale treatments designed for a population of one.
Because these are ultra-small, one-off trials rather than the large controlled studies regulators typically rely on, oversight and long-term safety data may be lagging behind the pace of new treatments.
What's your take?
Should governments and hospitals fast-track funding for personalized, one-patient gene-editing therapies like the one built for baby KJ Muldoon? Yes 
No 
Other 
Sources:
#CRISPR #GeneEditing #PrecisionMedicine #Bioethics
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