(Q30882233)

27 June 2017

title

Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs (English)

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27 June 2017

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Timothy Nichols

1

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27 June 2017

Margaret H Whitford

2

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27 June 2017

Valder R Arruda

3

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27 June 2017

Hansell Hall Stedman

4

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27 June 2017

Mark A Kay

5

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27 June 2017

Katherine A High

6

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27 June 2017

language of work or name

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30 December 2014

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Human gene therapy. Clinical development

27 June 2017

published in

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Canine hemophilia B resulting from a point mutation with unusual consequences

27 June 2017

cites work

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12 December 2020

A deletion mutation causes hemophilia B in Lhasa Apso dogs

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12 December 2020

Toxicity and morbidity of isolated limb perfusion

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12 December 2020

Isolated limb infusion with cytotoxic agents: a simple alternative to isolated limb perfusion

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

1 reference

12 December 2020

The human factor IX gene as germline mutagen test: samples from Mainland China have the putatively endogenous pattern of mutation

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12 December 2020

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

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12 December 2020

Absence of circulating factor IX antigen in hemophilia B dogs of the UNC-Chapel Hill colony

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12 December 2020

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

Aberrant splicing and premature termination of transcription of the FVIII gene as a cause of severe canine hemophilia A: similarities with the intron 22 inversion mutation in human hemophilia.

1 reference

12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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.

1 reference

12 December 2020

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

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12 December 2020

Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B.

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12 December 2020

Pathways of cell infection by parvoviruses and adeno-associated viruses

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12 December 2020

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

1 reference

12 December 2020

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

1 reference

12 December 2020

Molecular genotyping of the Italian cohort of patients with hemophilia B.

1 reference

12 December 2020

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

1 reference

12 December 2020

Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector

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12 December 2020

Large animal models and gene therapy

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12 December 2020

Glanzmann's thrombasthenia treatment: a prospective observational registry on the use of recombinant human activated factor VII and other hemostatic agents

1 reference

12 December 2020

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

1 reference

12 December 2020

Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

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12 December 2020

Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy

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12 December 2020

CD8(+) T-cell responses to adeno-associated virus capsid in humans

1 reference

12 December 2020

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression

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12 December 2020

Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer

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12 December 2020

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.

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12 December 2020

AAV vector integration sites in mouse hepatocellular carcinoma.

1 reference

12 December 2020

Adverse reproductive outcomes in urban women with adeno-associated virus-2 infections in early pregnancy

1 reference

12 December 2020

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

1 reference

12 December 2020

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

1 reference

12 December 2020

Review of companion animal viral diseases and immunoprophylaxis

1 reference

12 December 2020

Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency

1 reference

12 December 2020

X-linked thrombophilia with a mutant factor IX (factor IX Padua).

1 reference

12 December 2020

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

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12 December 2020

Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs.

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12 December 2020

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

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12 December 2020

Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.

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12 December 2020

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

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12 December 2020

Assessing the potential for AAV vector genotoxicity in a murine model

1 reference

12 December 2020

Cell phones and landlines: the impact of gene therapy on the cost and availability of treatment for hemophilia

1 reference

12 December 2020

Immune Responses to rAAV6: The Influence of Canine Parvovirus Vaccination and Neonatal Administration of Viral Vector

1 reference

12 December 2020

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

1 reference

12 December 2020

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

1 reference

12 December 2020

Spectrum of F9 mutations in Chinese haemophilia B patients: identification of 20 novel mutations

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12 December 2020

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

1 reference

12 December 2020

Gene therapy for hemophilia: addressing the coming challenges of affordability and accessibility

1 reference

12 December 2020

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

1 reference

12 December 2020

Ledipasvir and sofosbuvir for untreated HCV genotype 1 infection

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12 December 2020

Current status of haemophilia gene therapy

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12 December 2020

The special case of gene therapy pricing

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12 December 2020

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

1 reference

12 December 2020

Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial

1 reference

12 December 2020

Identifiers

DOI

10.1089/HUM.2014.153

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27 June 2017

25675273

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