An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

The baby boy with a rare urea cycle disorder received the bespoke therapy within a few months of birth and is doing well, ...

The baby boy received the bespoke therapy within a few months of birth and is doing well, according to a presentation at ASGCT.

Treated with an individualized gene-editing therapy that corrects mutations directly on the genome, a patient shows ...