Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors. - Wikidata - wikimedia - TriplyDB

Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.

Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.

http://www.wikidata.org/entity/Q34621668

about

Megakaryocyte- and megakaryocyte precursor-related gene therapies The gene therapy journey for hemophilia: are we there yet?Animal models of hemophilia Gene therapy for hemophilia Lessons Learned from Animal Models of Inherited Bleeding Disorders.Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery Portal vein delivery of viral vectors for gene therapy for hemophilia.Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.A novel cell-sheet technology that achieves durable factor VIII delivery in a mouse model of hemophilia A Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates.Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice Light chain of factor VIII is sufficient for accelerating cleavage of von Willebrand factor by ADAMTS13 metalloprotease.Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A Basic FGF or VEGF gene therapy corrects insufficiency in the intrinsic healing capacity of tendons.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.Engineering Factor Viii for Hemophilia Gene Therapy.Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype.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.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.Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A.Pharmacokinetics and ex vivo whole blood clot formation of a new recombinant FVIII (N8) in haemophilia A dogs.Pharmacokinetics and pharmacodynamics of turoctocog alfa and N8-GP in haemophilia A dogs Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A.Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice.Endogenous migration modulators as parent compounds for the development of novel cardiovascular and anti-inflammatory drugs.Recent advances in gene therapy for lysosomal storage disorders.Animal medical genetics: a perspective on the epidemiology and control of inherited disorders.Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection.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.Potential for cellular stress response to hepatic factor VIII expression from AAV vector Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia.

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Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.

http://www.wikidata.org/entity/Q34621668

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2010 nî lūn-bûn

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2010 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած

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2010年の論文

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Molecular Therapy

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Amy M Lange

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Denise E Sabatino

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Ekaterina S Altynova

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Elizabeth P Merricks

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Haig H Kazazian

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Helen G Franck

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Rita Sarkar

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Shangzhen Zhou

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Timothy C Nichols

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Valder R Arruda

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P2860

Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps.

Adeno-associated virus vector integration

Association of the factor VIII light chain with von Willebrand factor

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model.

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model.

Adeno-associated virus vectors can be efficiently produced without helper virus.

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy.

Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.

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442-449

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10.1038/MT.2010.240

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