2022
DOI: 10.1182/blood-2022-170646
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Inhibiting Activated Protein C Cleavage of Factor VIII for Hemophilia a Gene Therapy

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“…39 More recently, in transgene expression studies the APC-resistant FVIII R336Q/R562Q mutant (termed AAV-FVIII QQ) appeared to exhibit 5-fold higher hemostatic function than that of the WT in vivo 14 and blood loss assessed in the tail-clip assay was 5-10-fold lower than AAV-FVIII-WT. 40 These results indicated that the FVIII mutant could achieve hemostatic efficacy at lower AAV vector doses. The FVIII mutant, D519V/E665V/K1813A, used in our experiments could offer an ~8-fold gain of function in coagulation potential and hemostatic efficacy relative to WT in the murine model, although the precise mechanism(s) how this mutation modulates A2 dissociation remain unclear.…”
Section: Discussionmentioning
confidence: 93%
“…39 More recently, in transgene expression studies the APC-resistant FVIII R336Q/R562Q mutant (termed AAV-FVIII QQ) appeared to exhibit 5-fold higher hemostatic function than that of the WT in vivo 14 and blood loss assessed in the tail-clip assay was 5-10-fold lower than AAV-FVIII-WT. 40 These results indicated that the FVIII mutant could achieve hemostatic efficacy at lower AAV vector doses. The FVIII mutant, D519V/E665V/K1813A, used in our experiments could offer an ~8-fold gain of function in coagulation potential and hemostatic efficacy relative to WT in the murine model, although the precise mechanism(s) how this mutation modulates A2 dissociation remain unclear.…”
Section: Discussionmentioning
confidence: 93%