Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice. - Wikidata - awgldk - TriplyDB

Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.

Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.

http://www.wikidata.org/entity/Q37292357

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Complexity of immune responses to AAV transgene products - Example of factor IX.A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice.Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A.Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice.Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.Gene Therapy for Hemophilia.Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver.

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Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.

http://www.wikidata.org/entity/Q37292357

description

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 28 September 2016

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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

Overexpression of factor VIII ...... r stress in hemophilia A mice.

@en

Overexpression of factor VIII ...... r stress in hemophilia A mice.

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type

label

Overexpression of factor VIII ...... r stress in hemophilia A mice.

@en

Overexpression of factor VIII ...... r stress in hemophilia A mice.

@nl

prefLabel

Overexpression of factor VIII ...... r stress in hemophilia A mice.

@en

Overexpression of factor VIII ...... r stress in hemophilia A mice.

@nl

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Molecular Therapy. Methods & Clinical Development

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Overexpression of factor VIII ...... r stress in hemophilia A mice.

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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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Giang N Nguyen

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P2860

In vivo efficacy of platelet-delivered, high specific activity factor VIII variants

Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A.

Murine coagulation factor VIII is synthesized in endothelial cells.

A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII.

In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection

Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery

The past and future of haemophilia: diagnosis, treatments, and its complications.

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

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

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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16064

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scholarly article

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10.1038/MTM.2016.64

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3

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2016-09-28T00:00:00Z

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27738645

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Coagulation factor VIII

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5040173

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