KJ, the first baby saved thanks to personalized gene therapy

“A turning point in the fifty-year quest to read and repair the code of life,” enthuses the medical news site STAT News . And this promise has the face of KJ Muldoon, a chubby, blue-eyed 9-and-a-half-month-old baby, the first to benefit from personalized gene therapy using the revolutionary Crispr technique.

Kiran Musunuru, a gene editing researcher at the University of Pennsylvania, revealed on Thursday, May 15, that his team had managed to “ penetrate the genome of this very sick little boy and rewrite the only error in his genetic material.” In other words, of the three billion letters in the human genome, a mutation in just one of the letters in this code was the cause of KJ’s life-threatening illness.

The announcement of this medical success was made at the annual meeting of the American Society for Gene and Cell Therapies, and The New England Journal of Medicine simultaneously published an article on the medical history of this little American, who has lived 24 hours a day at the Children's Hospital of Philadelphia since his birth.

It was “a few days after his premature birth that the medical team following him became worried