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

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

To Shakespeare’s Hamlet we humans are “the paragon of animals”. But recent advances in genetics are suggesting that humans ...

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

FVIII, the blood clotting protein missing in hemophilia A, adopts multiple shapes, making it more likely to be targeted by ...

Researchers from the Centenary Institute have uncovered a reason why some people with hemophilia A develop resistance to ...

Genentech’s short film draws on the experiences of multiple families caring for children with hemophilia A, seeking to ...

Cell and gene therapies harbor great hopes for biomedicine. They are becoming increasingly important for diseases that are ...

A man’s acquired hemophilia coincided with a rare liver disease, a connection that had not been reported before, per a case ...

MHH haematologists successfully treat seriously ill patients with acquired haemophilia A with CAR-T cells.

Jim Wilson‘s pioneering gene therapy explorations have helped launch eight companies focused on translating his 40 years of ...

Scientists working at the NIH’s BRAIN Initiative have created new viral vectors that one day could lead to gene therapies for ...