Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.
about
In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical applicationHemophilia A: an ideal disease to correct in uteroAnimal models of hemophiliaCurrent animal models of hemophilia: the state of the artLessons Learned from Animal Models of Inherited Bleeding Disorders.A review of current methods for assessing hemostasis in vivo and introduction to a potential alternative approach.ARFI ultrasound monitoring of hemorrhage and hemostasis in vivo in canine von Willebrand disease and hemophiliaTranslational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogsClinical and molecular characterization of a re-established line of sheep exhibiting hemophilia A.Gene therapy in large animal models of human genetic diseases. IntroductionRecombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model.Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy.Assessing the potential for AAV vector genotoxicity in a murine model.Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.Porcine and canine von Willebrand factor and von Willebrand disease: hemostasis, thrombosis, and atherosclerosis studies.Phenotypic correction of hemophilia A in sheep by postnatal intraperitoneal transplantation of FVIII-expressing MSC.Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal modelsSuccessful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.In vivo gene transfer strategies to achieve partial correction of von Willebrand disease.The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene DeliveryAdvanced therapies for the treatment of hemophilia: future perspectives.Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype.Animal models of hemophilia and related bleeding disorders.Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models.The Jeremiah Metzger Lecture: gene therapy for inherited disorders: from Christmas disease to Leber's amaurosis.Pharmacokinetics and ex vivo whole blood clot formation of a new recombinant FVIII (N8) in haemophilia A dogs.Pharmacokinetics, pharmacodynamics and safety of recombinant canine FVIIa in a study dosing one haemophilia A and one haemostatically normal dog.Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide.The function of dog models in developing gene therapy strategies for human health.Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies.Anti-factor IXa/X bispecific antibody ACE910 prevents joint bleeds in a long-term primate model of acquired hemophilia A.Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia.Improving miRNA Delivery by Optimizing miRNA Expression Cassettes in Diverse Virus Vectors.Liver-directed lentiviral gene therapy in a dog model of hemophilia B.Sensitivity of whole blood clotting time and activated partial thromboplastin time for factor IX: relevance to gene therapy and determination of post-transfusion elimination time of canine factor IX in hemophilia B dogs.Soy Phosphatidylinositol-Containing Lipid Nanoparticle Prolongs the Plasma Survival and Hemostatic Efficacy of B-domain-Deleted Recombinant Canine Factor VIII in Hemophilia A Dogs.Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A.Characterization of canine coagulation factor VII and its complex formation with tissue factor: canine-human cross-species compatibility.The expanding menagerie: animal models of hemophilia A.
P2860
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P2860
Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.
description
2009 nî lūn-bûn
@nan
2009 թուականի Յունուարին հրատարակուած գիտական յօդուած
@hyw
2009 թվականի հունվարին հրատարակված գիտական հոդված
@hy
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
name
Protein replacement therapy an ...... se, and factor VII deficiency.
@ast
Protein replacement therapy an ...... se, and factor VII deficiency.
@en
type
label
Protein replacement therapy an ...... se, and factor VII deficiency.
@ast
Protein replacement therapy an ...... se, and factor VII deficiency.
@en
prefLabel
Protein replacement therapy an ...... se, and factor VII deficiency.
@ast
Protein replacement therapy an ...... se, and factor VII deficiency.
@en
P2093
P2860
P921
P356
P1433
P1476
Protein replacement therapy an ...... se, and factor VII deficiency.
@en
P2093
Aaron M Dillow
Dwight A Bellinger
Elizabeth P Merricks
Helen W G Franck
Katherine A High
Robin A Raymer
Timothy C Nichols
Valder R Arruda
P2860
P304
P356
10.1093/ILAR.50.2.144
P577
2009-01-01T00:00:00Z