| scholarly article | Q13442814 |
| P50 | author | X. Long Zheng | Q56808910 |
| P2093 | author name string | R J Davidson | |
| R M Camire | |||
| V R Arruda | |||
| L A George | |||
| D E Sabatino | |||
| C B Zander | |||
| G N Nguyen | |||
| J I Siner | |||
| P2860 | cites work | Further characterization of factor VIII-deficient mice created by gene targeting: RNA and protein studies | Q45879427 |
| Construction and characterization of an active factor VIII variant lacking the central one-third of the molecule | Q45886577 | ||
| A new recombinant procoagulant protein derived from the cDNA encoding human factor VIII | Q67252653 | ||
| Arg-X-Lys/Arg-Arg motif as a signal for precursor cleavage catalyzed by furin within the constitutive secretory pathway | Q67991478 | ||
| Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells | Q70385746 | ||
| Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization | Q71735042 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| Transplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A mice | Q30481282 | ||
| Proteolytic requirements for thrombin activation of anti-hemophilic factor (factor VIII) | Q33565918 | ||
| Regulated cleavage of prothrombin by prothrombinase: repositioning a cleavage site reveals the unique kinetic behavior of the action of prothrombinase on its compound substrate | Q33581285 | ||
| Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model | Q34078730 | ||
| Factor VIII and platelets synergistically accelerate cleavage of von Willebrand factor by ADAMTS13 under fluid shear stress | Q34121211 | ||
| A zymogen-like factor Xa variant corrects the coagulation defect in hemophilia | Q34149960 | ||
| Adeno-associated virus vectors can be efficiently produced without helper virus | Q34479933 | ||
| A single chain variant of factor VIII Fc fusion protein retains normal in vivo efficacy but exhibits altered in vitro activity | Q34557556 | ||
| Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors | Q34621668 | ||
| Long-term safety and efficacy of factor IX gene therapy in hemophilia B. | Q34783922 | ||
| Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery | Q35184180 | ||
| Apoptotic effects of platelet factor VIII on megakaryopoiesis: implications for a modified human FVIII for platelet-based gene therapy | Q35306206 | ||
| Profile of efraloctocog alfa and its potential in the treatment of hemophilia A. | Q35565320 | ||
| Isolation and characterization of human factor VIII: molecular forms in commercial factor VIII concentrate, cryoprecipitate, and plasma | Q35603064 | ||
| Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant | Q36797248 | ||
| Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype | Q36873312 | ||
| Stabilization of factor VIII in plasma by the von Willebrand factor. Studies on posttransfusion and dissociated factor VIII and in patients with von Willebrand's disease | Q37046074 | ||
| Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A. | Q37403833 | ||
| Post-translational requirements for functional factor V and factor VIII secretion in mammalian cells | Q38306964 | ||
| Biosynthesis, assembly and secretion of coagulation factor VIII. | Q38337120 | ||
| Improved vectors for stable expression of foreign genes in mammalian cells by use of the untranslated leader sequence from EMC virus | Q40506219 | ||
| Removal of B-domain sequences from factor V rather than specific proteolysis underlies the mechanism by which cofactor function is realized | Q40581702 | ||
| Physicochemical characterisation of rVIII-SingleChain, a novel recombinant single-chain factor VIII. | Q40876006 | ||
| Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein | Q41611929 | ||
| Structural and functional characterization of Factor VIII-delta II, a new recombinant Factor VIII lacking most of the B-domain | Q41676536 | ||
| Thrombin cleavage analysis of a novel antihaemophilic factor variant, factor VIII delta II. | Q41695610 | ||
| Omental implantation of BOECs in hemophilia dogs results in circulating FVIII antigen and a complex immune response | Q42218725 | ||
| Preclinical efficacy and safety of rVIII-SingleChain (CSL627), a novel recombinant single-chain factor VIII. | Q44016990 | ||
| Coagulant properties of hybrid human/porcine factor VIII molecules. | Q44885273 | ||
| Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs | Q45856596 | ||
| Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment | Q45864060 | ||
| Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A | Q45868121 | ||
| Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. | Q45875215 | ||
| P433 | issue | 1 | |
| P921 | main subject | Coagulation factor VIII | Q410137 |
| hemophilia A | Q2092064 | ||
| hemophilia | Q134003 | ||
| gene therapy | Q213901 | ||
| furin cleavage site | Q129704795 | ||
| P304 | page(s) | 110-121 | |
| P577 | publication date | 2016-11-25 | |
| P1433 | published in | Journal of Thrombosis and Haemostasis | Q6296004 |
| P1476 | title | Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A | |
| P478 | volume | 15 |
| Q91753661 | Correction of bleeding in experimental severe hemophilia A by systemic delivery of factor VIII-encoding mRNA |
| Q64376622 | Evaluation of the activity levels of rat FVIII and human FVIII delivered by adeno-associated viral vectors both in vitro and in vivo |
| Q99555300 | Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing |
| Q47225946 | Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice |
| Q89769521 | Identification of Key Coagulation Activity Determining Elements in Canine Factor VIII |
| Q45866877 | Molecular therapy of primary hyperoxaluria |
| Q45875647 | Novel alternate hemostatic agents for patients with inhibitors: beyond bypass therapy. |
| Q54975244 | Platelet-Targeted Gene Therapy for Hemophilia. |
| Q61811175 | Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy |
| Q52431082 | Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9. |
| Q90595648 | Update on clinical gene therapy for hemophilia |
| Q88739439 | [Research advances on gene therapy for hemophilia] |
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