Gene therapy for the hemophilias (Q64377524)

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6 November 2019

review article

1 reference

title

Gene therapy for the hemophilias (English)

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6 November 2019

author name string

VandenDriessche T

series ordinal

1

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6 November 2019

Collen D

series ordinal

2

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6 November 2019

Chuah MK

series ordinal

3

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6 November 2019

language of work or name

English

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publication date

1 July 2003

1 reference

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Journal of Thrombosis and Haemostasis

6 November 2019

published in

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6 November 2019

volume

1

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6 November 2019

issue

7

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6 November 2019

page(s)

1550-1558

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Advances toward gene therapy for hemophilia at the millennium

6 November 2019

cites work

1 reference

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Gene therapy for hemophilia: hopes and hurdles

21 January 2018

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Gene therapy for the hemophilias

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

21 January 2018

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Expression of human factor VIII by splicing between dimerized AAV vectors

21 January 2018

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Induction of tolerance to human factor VIII in mice

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

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

21 January 2018

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

21 January 2018

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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

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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

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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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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

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

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

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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

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

21 January 2018

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

21 January 2018

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Persistent expression of canine factor IX in hemophilia B canines

21 January 2018

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

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

21 January 2018

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

21 January 2018

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Gene therapy for hemophilia

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

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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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

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

21 January 2018

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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

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

21 January 2018

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Efficient lentiviral transduction of liver requires cell cycling in vivo

21 January 2018

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

21 January 2018

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Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging.

21 January 2018

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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

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

21 January 2018

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

21 January 2018

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

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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Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors

21 January 2018

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

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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Site-specific genomic integration produces therapeutic Factor IX levels in mice

21 January 2018

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

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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Adenovirus-mediated factor VIII gene expression results in attenuated anti-factor VIII-specific immunity in hemophilia A mice compared with factor VIII protein infusion

21 January 2018

1 reference

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An innate sense of danger

21 January 2018

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Dangerous liaisons: the role of "danger" signals in the immune response to gene therapy

21 January 2018

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Murine leukemia induced by retroviral gene marking

21 January 2018

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Gene therapy for SCID--a complication after remarkable progress

21 January 2018

1 reference

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Chromosomal effects of adeno-associated virus vector integration

21 January 2018

1 reference

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Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

21 January 2018

1 reference

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10.1046/J.1538-7836.2003.00265.X

21 January 2018

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:12871290%20AND%20SRC:MED&resulttype=core&format=json

6 November 2019

1 reference