Overcoming Hemophilia A Gene Therapy Limitations with an Enhanced Function Factor VIII Variant
A. R. Sternberg, C. Martos-Rus, R. Davidson, X. Liu, L. George
This study shows an enhanced FVIII variant (FVIII-QQ) can normalize hemostasis at low expression levels in hemophilia A mice, potentially improving gene therapy durability and safety.
bioRxiv · May 16, 2024 · PreprintPharmacology & Toxicology