Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs (Q30891500)

25 September 2017

1 reference

title

Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs (English)

1 reference

25 September 2017

0 references

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6

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

author name string

Timothy C Nichols

1

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

Margaret H Whitford

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

Valder R Arruda

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

Hansell H Stedman

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

Mark A Kay

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

publication date

12 February 2015

1 reference

Human gene therapy. Clinical development

25 September 2017

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

volume

26

1 reference

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2 June 2020

issue

1

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2 June 2020

page(s)

5-14

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

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

2 June 2020

cites work

1 reference

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21 June 2018

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Current status of haemophilia gene therapy

21 June 2018

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21 June 2018

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

21 June 2018

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

21 June 2018

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

21 June 2018

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

21 June 2018

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

21 June 2018

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

21 June 2018

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21 June 2018

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

21 June 2018

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

21 June 2018

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

21 June 2018

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

21 June 2018

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

21 June 2018

1 reference

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

21 June 2018

1 reference

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

Review of companion animal viral diseases and immunoprophylaxis

21 June 2018

1 reference

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

21 June 2018

1 reference

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

21 June 2018

1 reference

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

21 June 2018

1 reference

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

21 June 2018

1 reference

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

21 June 2018

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Large animal models and gene therapy

21 June 2018

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

21 June 2018

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

21 June 2018

1 reference

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Pathways of cell infection by parvoviruses and adeno-associated viruses

21 June 2018

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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 June 2018

1 reference

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

21 June 2018

1 reference

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

21 June 2018

1 reference

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

Toxicity and morbidity of isolated limb perfusion

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

A deletion mutation causes hemophilia B in Lhasa Apso dogs

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

Canine hemophilia B resulting from a point mutation with unusual consequences

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

The special case of gene therapy pricing

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

AAV vector integration sites in mouse hepatocellular carcinoma.

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4442577

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

25 November 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25675273

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

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

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25675273

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

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

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25675273

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1089/HUMC.2014.153

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25675273%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

PubMed publication ID

25675273

1 reference