Published in N Engl J Med on October 22, 2009
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood (2013) 2.09
Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy. Blood (2014) 1.64
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. Blood (2015) 1.64
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy. Blood (2012) 1.48
Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies. EMBO Mol Med (2013) 1.17
Procoagulant activity in hemostasis and thrombosis: Virchow's triad revisited. Anesth Analg (2011) 1.17
The gene therapy journey for hemophilia: are we there yet? Blood (2012) 1.10
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. Hum Gene Ther (2015) 0.97
Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs. Hum Gene Ther Clin Dev (2015) 0.95
Gene therapy for hemophilia. Front Biosci (Landmark Ed) (2015) 0.94
Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy. Mol Ther (2014) 0.93
Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice. Mol Ther (2013) 0.87
Adeno-associated viral vectors for the treatment of hemophilia. Hum Mol Genet (2015) 0.85
Gene therapy for hemophilia: the clot thickens. Hum Gene Ther (2014) 0.84
Gene therapy for immune tolerance induction in hemophilia with inhibitors. J Thromb Haemost (2016) 0.84
Muscle Gene Therapy for Hemophilia. J Genet Syndr Gene Ther (2012) 0.82
Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo. Blood (2011) 0.81
Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy. Mol Ther (2017) 0.80
In silico profiling of deleterious amino acid substitutions of potential pathological importance in haemophlia A and haemophlia B. J Biomed Sci (2012) 0.79
Prophylactic efficacy of BeneFIX vs Alprolix in hemophilia B mice. Blood (2016) 0.78
Gene therapy in an era of emerging treatment options for hemophilia B. J Thromb Haemost (2015) 0.78
Obstacles and future of gene therapy for hemophilia. Expert Opin Orphan Drugs (2015) 0.77
Progress towards gene therapy for haemophilia B. Int J Hematol (2014) 0.77
Developments in the treatment of hemophilia B: focus on emerging gene therapy. Appl Clin Genet (2013) 0.77
Biological functions of fucose in mammals. Glycobiology (2017) 0.76
Moving forward toward a cure for hemophilia B. Mol Ther (2015) 0.76
Specific factor IX mRNA and protein features favor drug-induced readthrough over recurrent nonsense mutations. Blood (2017) 0.75
Genetically modified adipose tissue-derived stem/stromal cells, using simian immunodeficiency virus-based lentiviral vectors, in the treatment of hemophilia B. Hum Gene Ther (2013) 0.75
Prevalence of Factor IX-R338L (Factor IX Padua) in a cohort of patients with venous thromboembolism and mild elevation of factor IX levels. Thromb Res (2010) 0.75
Bioengineering coagulation factors for improved hemophilia treatments: Comment on: the mutation F309S increases FVIII secretion in human cell line. Rev Bras Hematol Hemoter (2016) 0.75
Hepatocyte Is a Sole Cell Type Responsible for the Production of Coagulation Factor IX In Vivo. Cell Med (2012) 0.75
Extravascular FIX and coagulation. Thromb J (2016) 0.75
Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med (2008) 17.21
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med (2006) 13.51
In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature (2011) 4.81
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood (2002) 4.60
Below-knee elastic compression stockings to prevent the post-thrombotic syndrome: a randomized, controlled trial. Ann Intern Med (2004) 3.74
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest (2003) 3.52
Residual venous thrombosis as a predictive factor of recurrent venous thromboembolism. Ann Intern Med (2002) 3.51
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood (2002) 3.45
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther (2008) 3.28
Assessing the potential for AAV vector genotoxicity in a murine model. Blood (2010) 2.48
Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther (2006) 2.33
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood (2007) 2.05
The risk of recurrent venous thromboembolism after discontinuing anticoagulation in patients with acute proximal deep vein thrombosis or pulmonary embolism. A prospective cohort study in 1,626 patients. Haematologica (2007) 2.00
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy. Blood (2008) 1.98
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood (2013) 1.98
Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. Hum Gene Ther (2002) 1.98
Low plasma levels of tissue factor pathway inhibitor in patients with congenital factor V deficiency. Blood (2008) 1.90
Residual platelet factor V ensures thrombin generation in patients with severe congenital factor V deficiency and mild bleeding symptoms. Blood (2009) 1.80
Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle. Mol Ther (2008) 1.63
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest (2009) 1.54
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy. Blood (2012) 1.48
Contraceptive pills induce an improvement in congenital hypoplasminogenemia in two unrelated patients with ligneous conjunctivitis. Thromb Haemost (2003) 1.46
Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency. ILAR J (2009) 1.43
Influence of proband's characteristics on the risk for venous thromboembolism in relatives with factor V Leiden or prothrombin G20210A polymorphisms. Blood (2013) 1.39
Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors. Mol Ther (2010) 1.38
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. Mol Ther (2010) 1.35
Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy. Blood (2010) 1.32
Impact of inherited thrombophilia on venous thromboembolism in children: a systematic review and meta-analysis of observational studies. Circulation (2008) 1.26
Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector. J Virol (2002) 1.19
Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model. Blood (2009) 1.18
Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII. J Clin Invest (2004) 1.17
Emerging role of regulatory T cells in gene transfer. Curr Gene Ther (2007) 1.14
Strategies to modulate immune responses: a new frontier for gene therapy. Mol Ther (2009) 1.14
A prospective study on the prevalence and risk factors for neonatal thrombocytopenia and platelet alloimmunization among 9332 unselected Brazilian newborns. Transfusion (2007) 1.13
A zymogen-like factor Xa variant corrects the coagulation defect in hemophilia. Nat Biotechnol (2011) 1.12
Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype. Blood (2013) 1.11
Factor IX variants improve gene therapy efficacy for hemophilia B. Blood (2004) 1.09
In vivo efficacy of platelet-delivered, high specific activity factor VIII variants. Blood (2010) 1.08
Follow-up of sickle cell disease patients with priapism treated by hydroxyurea. Am J Hematol (2004) 1.04
The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene. Mol Ther (2007) 1.03
Incidence of venous thromboembolism in asymptomatic family members who are carriers of factor V Leiden: a prospective cohort study. Blood (2002) 1.02
Host and vector-dependent effects on the risk of germline transmission of AAV vectors. Mol Ther (2009) 1.00
The thioredoxin specificity of Drosophila GPx: a paradigm for a peroxiredoxin-like mechanism of many glutathione peroxidases. J Mol Biol (2006) 1.00
Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs. Hum Gene Ther Clin Dev (2015) 0.95
Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene. Blood (2004) 0.94
Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial. Mol Ther (2006) 0.94
Effects of long-term administration of high-dose recombinant human antithrombin in immunosuppressed primate recipients of porcine xenografts. Transplantation (2005) 0.89
Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model. Hum Gene Ther (2011) 0.89
Endothelial, platelet, and tissue factor-bearing microparticles in cancer patients with and without venous thromboembolism. Thromb Res (2011) 0.89
Factor VIIa-antithrombin complexes in patients with arterial and venous thrombosis. Thromb Haemost (2010) 0.87
Soluble endothelial protein C receptor (sEPCR) levels and venous thromboembolism in carriers of two dysfunctional protein C variants. Thromb Res (2006) 0.86
Thrombophilia as a predictor of persistent residual vein thrombosis. Haematologica (2008) 0.85
An underestimated combination of opposites resulting in enhanced thrombotic tendency. Blood (2005) 0.85
Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes. Mol Ther (2013) 0.84
The PAI-1 gene 4G/5G polymorphism and deep vein thrombosis in patients with inherited thrombophilia. Clin Appl Thromb Hemost (2003) 0.84
Peculiar whole blood rotation thromboelastometry (Rotem) profile in 40 sideropenic anaemia patients. Thromb Haemost (2008) 0.84
The risk of recurrent venous thromboembolism in patients with inherited deficiency of natural anticoagulants antithrombin, protein C and protein S. Haematologica (2006) 0.83
Screening for thrombophilia and antithrombotic prophylaxis in pregnancy: Guidelines of the Italian Society for Haemostasis and Thrombosis (SISET). Thromb Res (2009) 0.83
Intracranial haemorrhage in the Italian population of haemophilia patients with and without inhibitors. Haemophilia (2011) 0.83
The protein C pathway in cancer metastasis. Thromb Res (2012) 0.83
Hypercoagulability in overweight and obese subjects who are asymptomatic for thrombotic events. Thromb Haemost (2014) 0.82
The incidence of venous thromboembolism in thrombophilic children: a prospective cohort study. Blood (2002) 0.82
Role of fibrinolytic and clotting parameters in the diagnosis of liver veno-occlusive disease after hematopoietic stem cell transplantation in a pediatric population. Thromb Haemost (2005) 0.81
Objectives and methodology: Guidelines of the Italian Society for Haemostasis and Thrombosis (SISET). Thromb Res (2009) 0.81
In vitro and in vivo effects of the carbon monoxide-releasing molecule, CORM-3, in the xenogeneic pig-to-primate context. Xenotransplantation (2009) 0.81
Isolation and characterization of an antifactor V antibody causing activated protein C resistance from a patient with severe thrombotic manifestations. Blood (2002) 0.81
Biodistribution and shedding of AAV vectors. Methods Mol Biol (2011) 0.81
Platelet glycoprotein Ibalpha polymorphisms modulate the risk for myocardial infarction. Thromb Haemost (2004) 0.80
Characterisation of the serotonin efflux induced by cytosolic Ca2+ and Na+ concentration increase in human platelets. Cell Physiol Biochem (2004) 0.80
Substantial immune suppression required in gene therapy for muscular dystrophy? Neuromuscul Disord (2008) 0.80
The clinical performance of a chemiluminescent immunoassay in detecting anti-cardiolipin and anti-β2 glycoprotein I antibodies. A comparison with a homemade ELISA method. Clin Chem Lab Med (2015) 0.80
High prevalence of inherited thrombophilia in 'presumed peri-neonatal' ischemic stroke. Eur J Haematol (2007) 0.80
Optimal duration of anticoagulation. Provoked versus unprovoked VTE and role of adjunctive thrombophilia and imaging tests. Thromb Haemost (2014) 0.80
Inherited thrombophilia and venous thromboembolism. Semin Thromb Hemost (2006) 0.80
Pharmacological anti-thrombotic prophylaxis after elective caesarean delivery in thrombophilia unscreened women: should maternal age have a role in decision making? J Perinat Med (2014) 0.80
Reference values for thromboelastometry (ROTEM®) in cynomolgus monkeys (Macaca fascicularis). Thromb Res (2010) 0.79
Effects of long-term administration of recombinant human protein C in xenografted primates. Transplantation (2011) 0.79