(Q37903924)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21781236%20AND%20SRC:MED&resulttype=core&format=json

8 February 2020

review article

1 reference

title

Gene therapy for haemophilia: a long and winding road (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21781236%20AND%20SRC:MED&resulttype=core&format=json

8 February 2020

0 references

0 references

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21781236%20AND%20SRC:MED&resulttype=core&format=json

8 February 2020

author name string

K A High

series ordinal

1

1 reference

25 August 2017

publication date

1 July 2011

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21781236%20AND%20SRC:MED&resulttype=core&format=json

Journal of Thrombosis and Haemostasis

8 February 2020

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21781236%20AND%20SRC:MED&resulttype=core&format=json

8 February 2020

volume

9 Suppl 1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21781236%20AND%20SRC:MED&resulttype=core&format=json

8 February 2020

page(s)

2-11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21781236%20AND%20SRC:MED&resulttype=core&format=json

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

8 February 2020

cites work

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Effect of gene therapy on visual function in Leber's congenital amaurosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Adenovirus-mediated transfer of human factor IX gene in immunodeficient and normal mice: evidence for prolonged stability and activity of the transgene in liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Canine hemophilia B resulting from a point mutation with unusual consequences

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Intracellular interaction of von Willebrand factor and factor VIII depends on cellular context: lessons from platelet-expressed factor VIII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

In vivo efficacy of platelet-delivered, high specific activity factor VIII variants

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Progress towards liver and lung-directed gene therapy with helper-dependent adenoviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.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.1538-7836.2011.04369.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.1538-7836.2011.04369.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.1538-7836.2011.04369.X

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 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.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.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Cellular immune response to cryptic epitopes during therapeutic gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.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.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Hemophilia gene therapy: a Holy Grail found

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Codon optimization of human factor VIII cDNAs leads to high-level expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

AAV vector integration sites in mouse hepatocellular carcinoma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Assessing the potential for AAV vector genotoxicity in a murine model

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Human immunodeficiency and hepatitis virus infections and their associated conditions and treatments among people with haemophilia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Large-scale analysis of adeno-associated virus vector integration sites in normal human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.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.1538-7836.2011.04369.X

Reduced bleeding events with subcutaneous administration of recombinant human factor IX in immune-tolerant hemophilia B dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.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.1538-7836.2011.04369.X

Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Analysis of the spatial and temporal characteristics of platelet-delivered factor VIII-based clots

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.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.1538-7836.2011.04369.X

Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Long-term expression of murine activated factor VII is safe, but elevated levels cause premature mortality

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Successful treatment of canine hemophilia by continuous expression of canine FVIIa

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X

Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1538-7836.2011.04369.X