Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A.
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Megakaryocyte- and megakaryocyte precursor-related gene therapiesProgress and challenges in the development of a cell-based therapy for hemophilia ACurrent animal models of hemophilia: the state of the artGene therapy for hemophiliaGene therapy in an era of emerging treatment options for hemophilia BLessons Learned from Animal Models of Inherited Bleeding Disorders.Murine coagulation factor VIII is synthesized in endothelial cells.In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infectionDevelopment of Gene Transfer for Induction of Antigen-specific Tolerance.Apoptotic effects of platelet factor VIII on megakaryopoiesis: implications for a modified human FVIII for platelet-based gene therapy.The important role of von Willebrand factor in platelet-derived FVIII gene therapy for murine hemophilia A in the presence of inhibitory antibodies.Donor antigen-primed regulatory T cells permit liver regeneration and phenotype correction in hemophilia A mouse by allogeneic bone marrow stem cells.von Willebrand factor propeptide: biology and clinical utilityIn situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCsThe immunogenicity of platelet-derived FVIII in hemophilia A mice with or without preexisting anti-FVIII immunityOverexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A.Current status of haemophilia gene therapy.Biological therapies for inherited diseases: social and bioethical considerations. Hemophilia as an example.Obstacles and future of gene therapy for hemophilia.New approaches to gene and cell therapy for hemophilia.Platelet-delivered therapeutics.Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies.Gene therapy for immune tolerance induction in hemophilia with inhibitors.Targeting expression to megakaryocytes and platelets by lineage-specific lentiviral vectors.Rate-limiting roles of the tenase complex of factors VIII and IX in platelet procoagulant activity and formation of platelet-fibrin thrombi under flow.The search for the origin of factor VIII synthesis and its impact on therapeutic strategies for hemophilia A.Comparison of platelet-derived and plasma factor VIII efficacy using a novel native whole blood thrombin generation assay.Targeting factor VIII expression to platelets for hemophilia A gene therapy does not induce an apparent thrombotic risk in mice.Novel approaches to hemophilia therapy: successes and challenges.Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A.Platelet-Targeted Gene Therapy for Hemophilia.Gene therapy for hemophilia: what does the future hold?An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs
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Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A.
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article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on January 2013
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Platelet-targeted gene therapy ...... is in dogs with haemophilia A.
@en
Platelet-targeted gene therapy ...... is in dogs with haemophilia A.
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type
label
Platelet-targeted gene therapy ...... is in dogs with haemophilia A.
@en
Platelet-targeted gene therapy ...... is in dogs with haemophilia A.
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prefLabel
Platelet-targeted gene therapy ...... is in dogs with haemophilia A.
@en
Platelet-targeted gene therapy ...... is in dogs with haemophilia A.
@nl
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Platelet-targeted gene therapy ...... is in dogs with haemophilia A.
@en
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Alan T Nurden
David A Wilcox
Dwight A Bellinger
Elizabeth Merricks
Eric S Jensen
Kenneth Cornetta
Paquita Nurden
Paula M Jacobi
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P2888
P356
10.1038/NCOMMS3773
P407
P577
2013-01-01T00:00:00Z