Pharmacology & ToxicologyNature CommunicationsPeer-reviewed
Overcoming Hemophilia A Gene Therapy Limitations with an Enhanced Function Factor VIII Variant
Anna R. Sternberg, Cristina Martos-Rus, Robert J. Davidson…·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.
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