(Q35824841)

10 August 2017

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title

Prospects for gene therapy of haemophilia (English)

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10 August 2017

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Nathwani AC

1

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10 August 2017

Davidoff AM

series ordinal

2

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10 August 2017

Tuddenham EG

series ordinal

3

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10 August 2017

publication date

1 July 2004

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10 August 2017

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10 August 2017

volume

10

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10 August 2017

issue

4

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10 August 2017

page(s)

309-318

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Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

10 August 2017

cites work

1 reference

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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

21 January 2018

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

21 January 2018

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Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo

21 January 2018

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Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver

21 January 2018

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High-Level Factor VIII Gene ExpressionIn VivoAchieved by Nonviral Liver-Specific Gene Therapy Vectors

21 January 2018

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

21 January 2018

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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

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Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion

21 January 2018

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

21 January 2018

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Efficient production of human FVIII in hemophilic mice using lentiviral vectors

21 January 2018

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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

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

21 January 2018

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Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

21 January 2018

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Inhibition of costimulation allows for repeated systemic administration of adenoviral vector in rhesus monkeys.

21 January 2018

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Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

21 January 2018

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Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

1 reference

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00926.X

Gene therapy insertional mutagenesis insights

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00926.X

Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer

21 January 2018

1 reference

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AAV serotype 2 vectors preferentially integrate into active genes in mice

21 January 2018

1 reference

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A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction

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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Gene therapy: trials and tribulations

21 January 2018

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Adenovirus-mediated expresssion of the murine ecotropic receptor facilitates transduction of human hematopoietic cells with an ecotropic retroviral vector.

21 January 2018

1 reference

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Gene therapy progress and prospects: adenoviral vectors

21 January 2018

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Immune responses to adenovirus and adeno-associated virus in humans.

21 January 2018

1 reference

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The hemophilias--from royal genes to gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00926.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.1365-2516.2004.00926.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

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00926.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2516.2004.00926.X

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.1365-2516.2004.00926.X

10.1111/J.1365-2516.2004.00926.X

21 January 2018

Identifiers

DOI

1 reference

10 August 2017

1 reference