BioengineeringBloodPeer-reviewed
Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapy
Yuji Kashiwakura, Yuto Nakajima, Kio Horinaka…·Tsukasa Ohmori
This paper develops engineered FVIII variants for hemophilia A gene therapy, showing improved secretion, coagulation activity, and efficacy at lower vector doses in mice and macaques.
3 citations