2025-06-10
22 分钟Welcome to The Inquiry from the BBC World Service.
I'm Tanya Beckett.
Each week, one question, four expert witnesses and an answer.
In August of last year,
a baby boy was born in a hospital in Philadelphia in the United States.
He looked healthy, but very quickly it became clear something was wrong.
He was sleeping too much and wasn't eating.
Blood tests showed that baby KJ had sky-high levels of ammonia in his body.
The root cause was his genes, or more particularly,
a specific gene mutation that didn't allow his liver to expel the ammonia.
Doctors and scientists entered a race to try to treat him before his condition led to a coma.
In a few short months,
they had designed and gained government approval for a gene editing drug tailored specifically to him.
Earlier this year, baby KJ received three infusions, each a month apart,
containing billions of microscopic gene editors that honed in on the mutation in his liver.
He became the first ever recipient of a gene therapy designed to treat a single person.
KJ will need to be monitored for the rest of his life,
but a few months after the treatment, his health improved beyond recognition.
So this week on The Inquiry, we're asking,
have we seen a breakthrough in treating genetic diseases?