A baby who is the first patient in the world to be successfully treated with customized CRISPR gene editing therapy is ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...

KJ Muldoon's life-threatening disorder, called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, was treated with ...

Baby KJ was successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing ...

KJ Muldoon, a 10-month-old baby diagnosed with a rare metabolic disease called CPS1 has been at the Children's Hospital of ...

After 307 days at the Children's Hospital of Philadelphia, a local baby who was born with a rare and usually fatal disorder ...

A CRISPR treatment seems to have been effective for a baby’s devastating disease, but it is not clear whether such bespoke therapies can be widely applied ...

The one-off treatment highlights the promise of a new type of gene editing and the challenges of using it to treat extremely rare genetic diseases.

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

Scientists were able to create a bespoke treatment for KJ Muldoon’s rare genetic disorder within six months. It could be a ...