(Q35670173)

9 August 2017

title

Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation (English)

1 reference

9 August 2017

0 references

0 references

David W Scott

1

1 reference

9 August 2017

Jay N Lozier

series ordinal

2

1 reference

9 August 2017

language of work or name

English

0 references

publication date

7 November 2011

1 reference

British Journal of Haematology

9 August 2017

published in

1 reference

9 August 2017

volume

156

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9 August 2017

issue

3

1 reference

9 August 2017

page(s)

295-302

1 reference

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

9 August 2017

cites work

1 reference

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Immune tolerance induction in haemophilia: evidence and the way forward

12 July 2018

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Gene therapy for haemophilia: a long and winding road.

12 July 2018

1 reference

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

12 July 2018

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State-of-the-art gene-based therapies: the road ahead

12 July 2018

1 reference

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In vivo expansion of regulatory T cells with IL-2/IL-2 mAb complexes prevents anti-factor VIII immune responses in hemophilia A mice treated with factor VIII plasmid-mediated gene therapy

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Ex vivo gene transfer and correction for cell-based therapies

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Hemophilia gene therapy: a Holy Grail found

12 July 2018

1 reference

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Regulation of T cell activation by TLR ligands

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Factor VIII A3 domain substitution N1922S results in hemophilia A due to domain-specific misfolding and hyposecretion of functional protein

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Effect of B-cell depletion using anti-CD20 therapy on inhibitory antibody formation to human FVIII in hemophilia A mice

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Regulation of immunity and autoimmunity by B cells.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Immunomodulation for inhibitors in hemophilia A: the important role of Treg cells

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Delivery of nucleic acid therapeutics by genetically engineered hematopoietic stem cells

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Novel hyperactive transposons for genetic modification of induced pluripotent and adult stem cells: a nonviral paradigm for coaxed differentiation.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Efficacy of gene therapy for X-linked severe combined immunodeficiency

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

20 years of gene therapy for SCID.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Regulation of autoreactive B cell responses to endogenous TLR ligands

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Progress and prospects: immune responses to viral vectors

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

A role for thrombin in the initiation of the immune response to therapeutic factor VIII.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Immune tolerance: what is unique about the liver

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

The innate immune response to DNA.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Correction of murine hemophilia A following nonmyeloablative transplantation of hematopoietic stem cells engineered to encode an enhanced human factor VIII variant using a safety-augmented retroviral vector

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Factor VIII inhibitors: risk factors and methods for prevention and immune modulation

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Primary prophylaxis in children with haemophilia.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Activation of natural regulatory T cells by IgG Fc-derived peptide "Tregitopes"

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A mice

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Immunomodulation of transgene responses following naked DNA transfer of human factor VIII into hemophilia A mice

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Correction of murine hemophilia A by hematopoietic stem cell gene therapy

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Gene therapy for hemophilia: an imperative to succeed

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Induction of tolerance to factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Clinical gene transfer studies for hemophilia A.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

The hemophilias--from royal genes to gene therapy

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Anaphylaxis in patients with hemophilia

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Nephrotic syndrome associated with hypocomplementemia in a 4-year-old boy with hemophilia B.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Immune tolerance for the treatment of factor VIII inhibitors--twenty years' 'bonn protocol'.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Intracellular retention of a factor VIII protein with an Arg2307-->Gln mutation as a cause of haemophilia A.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Induction of immunological tolerance by porcine liver allografts

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Complications of hemophilia A treatment: factor VIII inhibitors

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Rapamycin promotes expansion of functional CD4+CD25+FOXP3+ regulatory T cells of both healthy subjects and type 1 diabetic patients

12 July 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Immune tolerance induction in 31 children with haemophilia A: is ITI less successful in African Americans?

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Stem cell-derived erythroid cells mediate long-term systemic protein delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Gene therapy insertional mutagenesis insights

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3+ T regulatory cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Prophylaxis in bleeding disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Reduction of the immune response to factor VIII mediated through tolerogenic factor VIII presentation by immature dendritic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Intracellular accumulation of factor VIII induced by missense mutations Arg593-->Cys and Asn618-->Ser explains cross-reacting material-reduced haemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

B-cell-delivered gene therapy induces functional T regulatory cells and leads to a loss of antigen-specific effector cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Use of central venous catheters in children with haemophilia: one haemophilia treatment centre experience

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Anti-CD3 prevents factor VIII inhibitor development in hemophilia A mice by a regulatory CD4+CD25+-dependent mechanism and by shifting cytokine production to favor a Th1 response

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

High expression reduces an antibody response after neonatal gene therapy with B domain-deleted human factor VIII in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Nephrotic syndrome as a complication of immune tolerance in hemophilia B.

25 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

FVIII inhibitor IgG subclass and FVIII polypeptide specificity determined by immunoblotting

25 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3257353

Gene therapists urged to learn more immunology

25 September 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

Endocytic receptor for pro-coagulant factor VIII: Relevance to inhibitor formation

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2011.08925.X

FVIII production by human lung microvascular endothelial cells

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22055221

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

1 reference

https://pubmed.ncbi.nlm.nih.gov/22055221

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Study of complement-mediated anaphylaxis in humans. The role of IgG subclasses (IgG1 and/or IgG4) in the complement-activating capacity of immune complexes

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22055221

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1111/J.1365-2141.2011.08925.X

1 reference

9 August 2017

1 reference

9 August 2017

1 reference