Overcoming Hemophilia A Gene Therapy Limitations with an Enhanced Function Factor VIII Variant
Anna R. Sternberg, Cristina Martos-Rus, Robert J. Davidson, Xueyuan Liu, Lindsey A. 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.
Nature Communications · Aug 21, 2024Peer-reviewedPharmacology & Toxicology