First Personalized Gene Therapy Using Base Editing Shows Promising Results in Baby with Rare Disorder

For the first time, a child with a life-threatening hereditary disease has been treated by surgically precise gene therapy. However, the little "KJ" still has to hope for healing.

For the first time, a futuristic gene therapy has helped a sick baby. The new technique allowed doctors to modify the DNA of a baby...

International researchers have called the treatment a promising medical milestone.

How a custom-made gene therapy could save one baby's life

A baby born with a rare, life-threatening genetic disease is now thriving after receiving an experimental gene-editing treatment. He's one of the first to be successfully treated with a bespoke therapy targeting a tiny but deadly error in his DNA. His doctors hope the technology could one day help the estimated 350 million people worldwide with rare diseases.

A baby with a rare and incurable disease became the first patient in the world to benefit from personalized genetic therapy, a hopeful achievement for other pathologies, an American medical team announced on Thursday. KJ Muldoon, a plump-faced, blue-eyed, nine-month-old baby, was diagnosed shortly after being born with a very rare and severe metabolic disorder called "carbamil phosphate deficiency synthetase" or CPS1. This disease is caused by m…

CRISPR Fulfills Its Promise with First-Ever Personalized Gene-Editing Therapy

Baby KJ's bleak future is brighter as CRISPR breakthrough paves the way to treat rare genetic disorders.