Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant - Wikidata - awgldk - TriplyDB

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

http://www.wikidata.org/entity/Q36797248

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The interplay of post-translational modification and gene therapy Megakaryocyte- and megakaryocyte precursor-related gene therapies Developments in the treatment of hemophilia B: focus on emerging gene therapy Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors Gene therapy for hemophilia The European Hematology Association Roadmap for European Hematology Research: a consensus document Expression and characterization of a codon-optimized blood coagulation factor VIII.Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy.Genetic influences on plasma CFH and CFHR1 concentrations and their role in susceptibility to age-related macular degeneration Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery PCR-based detection of gene transfer vectors: application to gene doping surveillance.In utero therapy for congenital disorders using amniotic fluid stem cells.Development of Gene Transfer for Induction of Antigen-specific Tolerance.Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery.Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.In vivo genome editing of the albumin locus as a platform for protein replacement therapy Transgene-host cell interactions mediate significant influences on the production, stability, and function of recombinant canine FVIII Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A.The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo.Adeno-associated viral vectors for the treatment of hemophilia.Issues in pediatric haemophilia care A genome editing primer for the hematologist Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid.Evaluation of the biological differences of canine and human factor VIII in gene delivery: implications in human hemophilia treatment.Development of gene therapy for blood disorders: an update.Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models.Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies.Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A.Anti-CD20 as the B-Cell Targeting Agent in a Combined Therapy to Modulate Anti-Factor VIII Immune Responses in Hemophilia a Inhibitor Mice Enhancing the pharmaceutical properties of protein drugs by ancestral sequence reconstruction Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A.Clinical potential of gene therapy: towards meeting the demand.Current status of haemophilia gene therapy.Current management of the hemophilic child: a demanding interlocutor. Quality of life and adequate cost-efficacy analysis.Obstacles and future of gene therapy for hemophilia.New approaches to gene and cell therapy for hemophilia.Our journey to successful gene therapy for hemophilia B.

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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

http://www.wikidata.org/entity/Q36797248

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BLOOD-2012-10-462200

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Therapeutic levels of FVIII fo ...... ovel human factor VIII variant

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Amit C Nathwani

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Andrew M Davidoff

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Arthur W Nienhuis

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Catherine Y C Ng

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Cecilia Rosales

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Christopher L Morton

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Deepak Raj

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Doyoung Lee

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Edward G D Tuddenham

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Federico Mingozzi

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P2860

Codon usage limitation in the expression of HIV-1 envelope glycoprotein

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Efficacy, safety and tolerability of recombinant factor VIII (REFACTO) in patients with haemophilia A: interim data from a postmarketing surveillance study in Germany and Austria.

Structural and functional characteristics of the B-domain-deleted recombinant factor VIII protein, r-VIII SQ.

Characterization of genome integrity for oversized recombinant AAV vector.

Effect of genome size on AAV vector packaging.

Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.

Prophylactic infusion regimens in the management of hemophilia.

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

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Simon N. Waddington

Peter J Lenting

Olivier Christophe

Katherine A High

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235682529

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