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

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

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

http://www.wikidata.org/entity/Q34541550

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Clinical development of gene therapy: results and lessons from recent successes Animal models of hemophilia Porcine model of hemophilia A Current animal models of hemophilia: the state of the art Gene therapy for hemophilia Lessons Learned from Animal Models of Inherited Bleeding Disorders.Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.Development of Gene Transfer for Induction of Antigen-specific Tolerance.AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.piggyBac-mediated phenotypic correction of factor VIII deficiency Profile of efraloctocog alfa and its potential in the treatment of hemophilia A.Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.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 Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A.Adeno-associated viral vectors for the treatment of hemophilia.Engineering Factor Viii for Hemophilia Gene Therapy.Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.Immune responses to AAV vectors: overcoming barriers to successful gene therapy Animal models of hemophilia and related bleeding disorders.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.Pharmacokinetics and ex vivo whole blood clot formation of a new recombinant FVIII (N8) in haemophilia A dogs.Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.Gene therapy for haemophilia: a long and winding road.Immune tolerance induction in haemophilia: evidence and the way forward.Inhibitors in childhood hemophilia A: genetic and treatment-related risk factors for development and eradication.Will gene therapy trump factor treatment in hemophilia?Inhibitors - genetic and environmental factors.Obstacles and future of gene therapy for hemophilia.The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer.In vivo induction of regulatory T cells for immune tolerance in hemophilia.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.Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice Potential for cellular stress response to hepatic factor VIII expression from AAV vector Complexity of immune responses to AAV transgene products - Example of factor IX.

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

http://www.wikidata.org/entity/Q34541550

description

2010 nî lūn-bûn

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2010 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած

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2010 թվականի սեպտեմբերին հրատարակված գիտական հոդված

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2010年の論文

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2010年論文

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2010年論文

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2010年論文

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2010年論文

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2010年論文

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2010年论文

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David Lillicrap

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Denise E Sabatino

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Elizabeth P Merricks

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Haig H Kazazian

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Helen W G Franck

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Jonathan D Finn

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Julie M Crudele

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Margareth C Ozelo

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Shangzhen Zhou

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Timothy C Nichols

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P2860

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Effect of gene therapy on visual function in Leber's congenital amaurosis

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

The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion.

Aberrant splicing and premature termination of transcription of the FVIII gene as a cause of severe canine hemophilia A: similarities with the intron 22 inversion mutation in human hemophilia.

Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy.

A novel monoclonal antibody specific for canine CD25 (P4A10): selection and evaluation of canine Tregs

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

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

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10.1182/BLOOD-2010-06-288001

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46578988

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20876851

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Coagulation factor VIII

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3031380

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