Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B (Q21245486)

18 August 2022

Ashley T Martino

2

Ashley T

Martino

1 reference

18 August 2022

Irene Zolotukhin

3

Irene

Zolotukhin

1 reference

18 August 2022

Hildegund C J Ertl

4

Hildegund CJ

Ertl

1 reference

18 August 2022

Roland W Herzog

5

Roland W

Herzog

1 reference

Journal of Translational Medicine

18 August 2022

language of work or name

English

0 references

publication date

2014

0 references

published in

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volume

12

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issue

1

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page(s)

25

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cites work

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The gene therapy journey for hemophilia: are we there yet?

19 March 2017

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Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

12 September 2017

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Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice

12 September 2017

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IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression

12 September 2017

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CpG-depleted adeno-associated virus vectors evade immune detection.

12 September 2017

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Differential type I interferon-dependent transgene silencing of helper-dependent adenoviral vs. adeno-associated viral vectors in vivo

12 September 2017

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MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV

12 September 2017

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

12 September 2017

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

12 September 2017

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Innate Immune Responses to AAV Vectors

12 September 2017

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Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges

12 September 2017

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Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results

12 September 2017

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Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

12 September 2017

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Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

12 September 2017

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The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver

12 September 2017

1 reference

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RETRACTED: Activation of the NF-kappaB pathway by adeno-associated virus (AAV) vectors and its implications in immune response and gene therapy

12 September 2017

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Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

12 September 2017

1 reference

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High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines.

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

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

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

AAV vectors avoid inflammatory signals necessary to render transduced hepatocyte targets for destructive T cells

12 September 2017

1 reference

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GITR engagement preferentially enhances proliferation of functionally competent CD4+CD25+FoxP3+ regulatory T cells

12 September 2017

1 reference

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Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicity

12 September 2017

1 reference

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Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

12 September 2017

1 reference

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

12 September 2017

1 reference

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

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

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

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy

12 September 2017

1 reference

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

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Systemic protein delivery by muscle-gene transfer is limited by a local immune response

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

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.

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

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

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

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

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B.

12 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Characterization of tissue tropism determinants of adeno-associated virus type 1.

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Promoter attenuation in gene therapy: interferon-gamma and tumor necrosis factor-alpha inhibit transgene expression

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Measuring immune responses to recombinant AAV gene transfer

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Optimal Immunofluorescent Staining for Human Factor IX and Infiltrating T Cells following Gene Therapy for Hemophilia B.

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Nonredundant roles of IL-10 and TGF-β in suppression of immune responses to hepatic AAV-factor IX gene transfer

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver.

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

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

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

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

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

Interferon-alpha inhibits the murine cytomegalovirus immediate-early gene expression by down-regulating NF-kappa B activity

30 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3904690

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

30 May 2018

1 reference

https://api.crossref.org/works/10.1186%2F1479-5876-12-25

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1186%2F1479-5876-12-25

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1186%2F1479-5876-12-25

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/10.1186%2F1479-5876-12-25

Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene.

21 January 2018

1 reference

https://api.crossref.org/works/10.1186%2F1479-5876-12-25

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/10.1186%2F1479-5876-12-25

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/10.1186%2F1479-5876-12-25

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/10.1186%2F1479-5876-12-25

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/10.1186%2F1479-5876-12-25

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/10.1186%2F1479-5876-12-25

21 January 2018

Identifiers

DOI

10.1186/1479-5876-12-25

1 reference

https://scigraph.springernature.com/pub.10.1186/1479-5876-12-25

1 reference

9 September 2019

1 reference

1 reference

PubMed publication ID

24460861

1 reference