This May Be the Most Important Medical Story of the Decade

Dr. Coller directs the RNA Innovation Center at Johns Hopkins University.

When KJ Muldoon was born in the summer of 2024, his parents were told he had a disease so rare, it strikes about one in 1.3 million newborns. His condition, a severe deficiency of an enzyme known as CPS1, left his tiny body unable to properly break down protein, flooding his blood with toxins that could cause brain damage or death. A liver transplant could correct the problem, but KJ was too young and too fragile to undergo one. With each passing day, the risk of irreversible neurological damage grew.

What happened next may become the most important medical story of the decade. In just six months, a team at Children’s Hospital of Philadelphia and Penn Medicine........

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