Preclinical and clinical gene therapy for haemophilia (Q35915704)

11 August 2017

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title

Preclinical and clinical gene therapy for haemophilia (English)

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11 August 2017

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Chuah MK

1

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11 August 2017

Collen D

series ordinal

2

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11 August 2017

Vandendriessche T

series ordinal

3

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11 August 2017

publication date

1 October 2004

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11 August 2017

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11 August 2017

volume

10 Suppl 4

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11 August 2017

page(s)

119-125

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Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein

11 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Expression of human factor VIII by splicing between dimerized AAV vectors

21 January 2018

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https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Induction of tolerance to human factor VIII in mice

21 January 2018

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https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector

21 January 2018

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Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

21 January 2018

1 reference

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Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

21 January 2018

1 reference

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Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

21 January 2018

1 reference

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Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.

21 January 2018

1 reference

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Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

21 January 2018

1 reference

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Factors influencing the development of an anti-factor IX (FIX) immune response following administration of adeno-associated virus-FIX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

21 January 2018

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Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

21 January 2018

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Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9.

21 January 2018

1 reference

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Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

21 January 2018

1 reference

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Persistent expression of canine factor IX in hemophilia B canines

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

21 January 2018

1 reference

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Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy

21 January 2018

1 reference

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Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Gene therapy for hemophilia: hopes and hurdles

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Persistent delivery of factor IX in mice: gene therapy for hemophilia using implantable microcapsules

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Nanoparticles for the delivery of genes and drugs to human hepatocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Use of blood outgrowth endothelial cells for gene therapy for hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

Efficient lentiviral transduction of liver requires cell cycling in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00984.X

10.1111/J.1365-2516.2004.00984.X

21 January 2018

Identifiers

DOI

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release_jrybe33z3bgjzalaku3ccapymy

11 August 2017

Fatcat ID

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Fatcat

https://api.fatcat.wiki/v0/release/jrybe33z3bgjzalaku3ccapymy

mapped directly with Wikidata item

24 November 2022

based on heuristic

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