Gene therapy in an era of emerging treatment options for hemophilia B (Q28087557)

language of work or name

1 reference

June 2015

1 reference

Journal of Thrombosis and Haemostasis

published in

1 reference

volume

13 Suppl 1

1 reference

page(s)

S151-60

1 reference

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

cites work

1 reference

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In vivo genome editing restores haemostasis in a mouse model of haemophilia

20 March 2017

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

20 March 2017

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

7 April 2017

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Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.

29 September 2017

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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

29 September 2017

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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

29 September 2017

1 reference

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

29 September 2017

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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

29 September 2017

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Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.

29 September 2017

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Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs

29 September 2017

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Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A.

29 September 2017

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Evidence of clinically significant extravascular stores of factor IX.

29 September 2017

1 reference

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Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

29 September 2017

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

29 September 2017

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Robust ZFN-mediated genome editing in adult hemophilic mice.

29 September 2017

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Molecular approaches for improved clotting factors for hemophilia

29 September 2017

1 reference

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Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice

29 September 2017

1 reference

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Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Prevalent inhibitors in haemophilia B subjects enrolled in the Universal Data Collection database

29 September 2017

1 reference

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy

29 September 2017

1 reference

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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.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Adeno-associated virus vectorology, manufacturing, and clinical applications

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Adeno-associated virus antibody profiles in newborns, children, and adolescents

29 September 2017

1 reference

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Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

29 September 2017

1 reference

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Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors

29 September 2017

1 reference

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Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial

29 September 2017

1 reference

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Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

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

29 September 2017

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

29 September 2017

1 reference

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Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX.

29 September 2017

1 reference

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Identification of a heparin-binding motif on adeno-associated virus type 2 capsids

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Identification of the endothelial cell binding site for factor IX.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Neutralizing antibodies against AAV2, AAV5 and AAV8 in healthy and HIV-1-infected subjects in China: implications for gene therapy using AAV vectors

2 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Overcoming preexisting humoral immunity to AAV using capsid decoys

2 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

A coagulation factor IX-deficient mouse model for human hemophilia B.

2 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4712690

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

2 June 2018

1 reference

Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

21 January 2018

1 reference

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

Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice

21 January 2018

1 reference

Neutralizing antibodies against adeno-associated viruses in inflammatory bowel disease patients: implications for gene therapy

21 January 2018

1 reference

Multicentre, randomized, open-label study of on-demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects

21 January 2018

1 reference

Long-acting recombinant factor IX Fc fusion protein (rFIXFc) for perioperative management of subjects with haemophilia B in the phase 3 B-LONG study

21 January 2018

1 reference

Risk factors for infection with HBV and HCV in a largecohort of hemophiliac males

21 January 2018

1 reference

Gene therapy for hemophilia

21 January 2018

1 reference

https://pubmed.ncbi.nlm.nih.gov/26149016

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Changing residue 338 in human factor IX from arginine to alanine causes an increase in catalytic activity

1 reference

https://pubmed.ncbi.nlm.nih.gov/26149016

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs

1 reference

https://pubmed.ncbi.nlm.nih.gov/26149016