The gene therapy journey for hemophilia: are we there yet? (Q26823818)

Crossref

language of work or name

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29 November 2012

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120

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issue

23

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page(s)

4482-7

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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

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

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Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

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

28 September 2017

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

28 September 2017

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

28 September 2017

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Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide

28 September 2017

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A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8.

28 September 2017

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

28 September 2017

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

28 September 2017

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Assessing the potential for AAV vector genotoxicity in a murine model

28 September 2017

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

28 September 2017

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

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

28 September 2017

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

28 September 2017

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

28 September 2017

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Host and vector-dependent effects on the risk of germline transmission of AAV vectors

28 September 2017

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

28 September 2017

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Manufacturing and characterizing AAV-based vectors for use in clinical studies

28 September 2017

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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

28 September 2017

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

28 September 2017

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Quantification of adeno-associated virus particles and empty capsids by optical density measurement

28 September 2017

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

28 September 2017

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Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

28 September 2017

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Sustained expression of human factor VIII in mice using a parvovirus-based vector.

28 September 2017

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Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein

28 September 2017

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Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver

28 September 2017

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Adeno-associated virus vector integration junctions.

28 September 2017

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

28 September 2017

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Alternative strategies for gene therapy of hemophilia

31 May 2018

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

31 May 2018

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

31 May 2018

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Activated factor VII: my story

31 May 2018

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

28 November 2018

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

28 November 2018

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Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

28 November 2018

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AAV vector integration sites in mouse hepatocellular carcinoma.

28 November 2018

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

28 November 2018

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In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance.

28 November 2018

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Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial

28 November 2018

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

28 November 2018

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

28 November 2018

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Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

28 November 2018

1 reference

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Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males

28 November 2018

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

28 November 2018

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Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

28 November 2018

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Trial halted after gene shows up in semen

28 November 2018

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