Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients? (Q36230845)

12 August 2017

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title

Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients? (English)

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12 August 2017

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High K

1

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12 August 2017

1 August 2005

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Journal of Thrombosis and Haemostasis

12 August 2017

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12 August 2017

3

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12 August 2017

8

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12 August 2017

1682-1691

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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

12 August 2017

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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.1538-7836.2005.01460.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.1538-7836.2005.01460.X

Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Gene therapy restores vision in a canine model of childhood blindness

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Long-term lowering of plasma cholesterol levels in LDL-receptor-deficient WHHL rabbits by gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Improved system for helper-dependent adenoviral vector production

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

A Line 1 insertion in the Factor IX gene segregates with mild hemophilia B in dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Toxicity of a first-generation adenoviral vector in rhesus macaques

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Cause of the 'inhibitor' phenotype in the haemophilias

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.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.1538-7836.2005.01460.X

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.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.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.1538-7836.2005.01460.X

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

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.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.1538-7836.2005.01460.X

Progress and problems with the use of viral vectors for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.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.1538-7836.2005.01460.X

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Efficient lentiviral transduction of liver requires cell cycling in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Current uses of isolated limb perfusion in the clinic and a model system for new strategies

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Adeno-associated virus vectors can be efficiently produced without helper virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.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.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Targeting survival: integration site selection by retroviruses and LTR-retrotransposons

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

HIV-1 integration in the human genome favors active genes and local hotspots

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Transcription start regions in the human genome are favored targets for MLV integration

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Characterization of the chicken beta-globin insulator

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

The emerging fields of suicide gene therapy and virotherapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.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.1538-7836.2005.01460.X

A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Persistent expression of canine factor IX in hemophilia B canines

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Effective gene therapy for an inherited CNS disease in a large animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

Treatment of canine cyclic neutropenia by lentivirus-mediated G-CSF delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2005.01460.X

10.1111/J.1538-7836.2005.01460.X

21 January 2018

Identifiers

DOI

1 reference

12 August 2017

1 reference

PubMed ID

16102034