Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors. (Q36085709)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

title

Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

0 references

0 references

0 references

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

author name string

L Wang

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

J-P Louboutin

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

P Bell

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

J A Greig

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

Y Li

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

D Wu

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

language of work or name

1 reference

1 October 2011

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

Journal of Thrombosis and Haemostasis

12 February 2020

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

volume

9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

issue

10

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

page(s)

2009-2019

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

Role of regulatory T cells in tolerance to coagulation factors

12 February 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Hepatic gene transfer as a means of tolerance induction to transgene products

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Induction of immune tolerance to FIX following muscular AAV gene transfer is AAV-dose/FIX-level dependent

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Enhancing transduction of the liver by adeno-associated viral vectors

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Recombinant adeno-associated virus vectors induce functionally impaired transgene product-specific CD8+ T cells in mice

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

New recombinant serotypes of AAV vectors.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Clades of Adeno-associated viruses are widely disseminated in human tissues.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Immunity to adenovirus and adeno-associated viral vectors: implications for gene therapy

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Gene therapy vectors based on adeno-associated virus type 1.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

A factor IX-deficient mouse model for hemophilia B gene therapy

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Persistent expression of canine factor IX in hemophilia B canines

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

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

27 July 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.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.1538-7836.2011.04491.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.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.1538-7836.2011.04491.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.1538-7836.2011.04491.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.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.1538-7836.2011.04491.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.1538-7836.2011.04491.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.1538-7836.2011.04491.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Efficient induction of immune tolerance to coagulation factor IX following direct intramuscular gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia a dogs and mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Phenotype correction of hemophilia A mice with adeno-associated virus vectors carrying the B domain-deleted canine factor VIII gene.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Major role of local immune responses in antibody formation to factor IX in AAV gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

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

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Gene transfer into skeletal muscle using novel AAV serotypes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Vaccines based on novel adeno-associated virus vectors elicit aberrant CD8+ T-cell responses in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04491.X

Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

Human factor IX corrects the bleeding diathesis of mice with hemophilia B.

23 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393098

10.1111/J.1538-7836.2011.04491.X

23 September 2018

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21883883%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

1 reference