ArticleThe Journal of clinical investigation2004
Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII.
Article in The Journal of clinical investigation, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
29 citing papers in PubMed, 64 citations in OpenAlex.
- Cure of Congenital Purpura Fulminans via Expression of Engineered Protein C Through Neonatal Genome Editing in Mice.Arteriosclerosis, thrombosis, and vascular biology · 2024Article
- The Development and Characterization of an scFv-Fc Fusion-Based Gene Therapy to Reduce the Psychostimulant Effects of Methamphetamine Abuse.The Journal of pharmacology and experimental therapeutics · 2020Article
- Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy.Molecular therapy. Methods & clinical development · 2019Review
- Gene-based FVIIa prophylaxis modulates the spontaneous bleeding phenotype of hemophilia A rats.Blood advances · 2019Article
- Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models.JCI insight · 2016Article
- Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII.Blood · 2016Article
- A chimeric platelet-targeted urokinase prodrug selectively blocks new thrombus formation.The Journal of clinical investigation · 2016Article
- Gene therapy for hemophilia.Frontiers in bioscience (Landmark edition) · 2015Review
- The endothelial protein C receptor enhances hemostasis of FVIIa administration in hemophilic mice in vivo.Blood · 2014Article
- Lessons Learned from Animal Models of Inherited Bleeding Disorders.Hematology education. European Hematology Association. Congress. Education Program · 2014Article
- Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
- Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease.Gene therapy · 2012Article
- Animal models of hemophilia.Progress in molecular biology and translational science · 2012Review
- Engineering Factor Viii for Hemophilia Gene Therapy.Journal of genetic syndromes & gene therapy · 2011Article
- Pharmacokinetics, pharmacodynamics and safety of recombinant canine FVIIa in a study dosing one haemophilia A and one haemostatically normal dog.Haemophilia : the official journal of the World Federation of Hemophilia · 2011Article
- A zymogen-like factor Xa variant corrects the coagulation defect in hemophilia.Nature biotechnology · 2011Article
- Article
- Proteases as therapeutics.The Biochemical journal · 2011Review
- Expression of recombinant human coagulation factor VII by the Lizard Leishmania expression system.Journal of biomedicine & biotechnology · 2011Article
- Long-term expression of canine FVIIa in hemophilic dogs.Thrombosis research · 2010Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
Hemophilia is a bleeding disorder caused by mutations in the genes encoding coagulation Factor VIII (FVIII) or FIX. Current treatment is through intravenous infusion of the missing protein. The major complication of treatment is the development of neutralizing Ab's to the clotting factor. Infusion of recombinant activated human Factor VII (rhFVIIa), driving procoagulant reactions independently of human FVIII (hFVIII) or hFIX, has been successful in such patients and could in theory provide hemostasis in all hemophilia patients. However, its high cost and short half-life have limited its use. Here, we report a novel treatment strategy with a recombinant adeno-associated virus vector delivering a modified FVII transgene that can be intracellularly processed and secreted as activated FVII (FVIIa). We show long-term expression, as well as phenotypic correction of hemophilia B mice following gene transfer of the murine FVIIa homolog, with no evidence of thrombotic complications at these doses. These data hold promise for a potential treatment for hemophilia and other bleeding disorders.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.