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

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

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

CRISPR-based gene editing therapy has been used for the first time to treat CPS1 deficiency, which promises a new era for ...

Baby KJ Muldoon was born with a rare genetic disorder called CPS1 deficiency and spent nearly the entire first year of his ...

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

KJ Muldoon, an infant with a life-threatening metabolic disorder who received a pioneering gene-editing therapy at Children’s Hospital of Philadelphia, went home Tuesday after 307 days at the ...

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

A child born with a life-threatening genetic disorder has been successfully treated using pioneering ethical gene therapy.

Scientists have used personalised gene-editing therapy to successfully treat a baby with a rare genetic condition. Could this ...

This may now be changing, after the first successful genetic treatment of an infant with CPS1 deficiency. Carbamoyl phosphate synthetase I (CPS1) is an enzyme that is crucial for breaking down the ...