Published in Blood on December 01, 2011
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood (2013) 1.36
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Mechanistic insights into the enhancement of adeno-associated virus transduction by proteasome inhibitors. J Virol (2013) 0.82
Kidney-specific expression of GFP by in-utero delivery of pseudotyped adeno-associated virus 9. Mol Ther Methods Clin Dev (2014) 0.81
In utero therapy for congenital disorders using amniotic fluid stem cells. Front Pharmacol (2014) 0.79
Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies. Gene Ther (2015) 0.77
Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII. Blood (2015) 0.76
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. Mol Ther Methods Clin Dev (2016) 0.75
In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model. Mol Ther (2017) 0.75
Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med (2008) 15.30
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med (2006) 13.51
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther (2003) 4.92
Treatment of rheumatoid arthritis by selective inhibition of T-cell activation with fusion protein CTLA4Ig. N Engl J Med (2003) 4.67
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med (1999) 4.61
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther (2009) 3.93
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A (2009) 3.58
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med (1999) 3.39
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. Mol Ther (2011) 3.10
In utero gene therapy rescues vision in a murine model of congenital blindness. Mol Ther (2004) 2.56
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
Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood (2005) 2.12
Long-term luciferase expression monitored by bioluminescence imaging after adeno-associated virus-mediated fetal gene delivery in rhesus monkeys (Macaca mulatta). Hum Gene Ther (2010) 1.80
Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood (2003) 1.75
Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. Blood (2001) 1.74
Gene delivery to the juvenile mouse liver using AAV2/8 vectors. Mol Ther (2008) 1.73
Factor VII Deficiency. Semin Thromb Hemost (2009) 1.69
Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood (2010) 1.68
Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep. Hum Gene Ther (2011) 1.61
Design and packaging of adeno-associated virus gene targeting vectors. J Virol (2000) 1.54
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther (2004) 1.54
Epidemiology of coagulation disorders. Baillieres Clin Haematol (1992) 1.53
Multiparameter RNA and codon optimization: a standardized tool to assess and enhance autologous mammalian gene expression. PLoS One (2011) 1.46
AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease. Mol Ther (2005) 1.41
Factor VII deficiency: defining the clinical picture and optimizing therapeutic options. Haemophilia (2008) 1.41
Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Blood (2004) 1.30
Enhancing transduction of the liver by adeno-associated viral vectors. Gene Ther (2008) 1.24
Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency. Atheroscler Suppl (2010) 1.22
Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation. Hum Gene Ther (2006) 1.21
Comparison of two codon optimization strategies to enhance recombinant protein production in Escherichia coli. Microb Cell Fact (2011) 1.20
In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor. Blood (2002) 1.16
Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol Ther (2005) 1.14
Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Ther (2006) 1.10
AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). Mol Ther (2011) 1.07
Adeno-associated virus gene transfer to mouse retina. Hum Gene Ther (1998) 1.06
Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease. Development (2004) 1.04
Generation of genetically-altered mice producing very low levels of coagulation factorVII. Thromb Haemost (2005) 1.00
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques. Mol Ther (2011) 1.00
Gender differences in serotype 2 adeno-associated virus biodistribution after administration to rodent salivary glands. Hum Gene Ther (2007) 0.95
Congenital factor VII deficiency: therapy with recombinant activated factor VII -- a critical appraisal. Haemophilia (2006) 0.95
Sexually dimorphic patterns of episomal rAAV genome persistence in the adult mouse liver and correlation with hepatocellular proliferation. Mol Ther (2009) 0.93
Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Hum Gene Ther (2005) 0.93
Intrahepatic injection of recombinant adeno-associated virus serotype 2 overcomes gender-related differences in liver transduction. Hum Gene Ther (2006) 0.91
Adeno-associated virus vector-mediated systemic delivery of IFN-beta combined with low-dose cyclophosphamide affects tumor regression in murine neuroblastoma models. Clin Cancer Res (2005) 0.87
Gene therapy in haemophilia--going for cure? Haemophilia (2010) 0.85
Factor VII deficiency and its treatment in delivery with recombinant factor VII. Eur J Obstet Gynecol Reprod Biol (2004) 0.79
Liver transplantation for factor VII deficiency. Transplantation (2001) 0.77
Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med (2008) 15.30
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med (2011) 11.01
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med (2006) 9.68
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest (2008) 7.05
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med (2002) 6.25
Mutations in VKORC1 cause warfarin resistance and multiple coagulation factor deficiency type 2. Nature (2004) 5.95
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet (2004) 5.43
The pediatric preclinical testing program: description of models and early testing results. Pediatr Blood Cancer (2007) 5.28
Adenovirus serotype 5 hexon mediates liver gene transfer. Cell (2008) 5.00
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med (2010) 4.76
Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res (2006) 3.63
Endotoxemia and sepsis mortality reduction by non-anticoagulant activated protein C. J Exp Med (2007) 3.35
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest (2007) 3.28
A robust model for read count data in exome sequencing experiments and implications for copy number variant calling. Bioinformatics (2012) 3.10
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. Mol Ther (2011) 3.10
Initial testing of the aurora kinase A inhibitor MLN8237 by the Pediatric Preclinical Testing Program (PPTP). Pediatr Blood Cancer (2010) 2.99
Bevacizumab-induced transient remodeling of the vasculature in neuroblastoma xenografts results in improved delivery and efficacy of systemically administered chemotherapy. Clin Cancer Res (2007) 2.97
Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med (2014) 2.92
Exosomes released from macrophages infected with intracellular pathogens stimulate a proinflammatory response in vitro and in vivo. Blood (2007) 2.92
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest (2007) 2.80
Essential role of the NADPH oxidase subunit p47(phox) in endothelial cell superoxide production in response to phorbol ester and tumor necrosis factor-alpha. Circ Res (2002) 2.79
Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood (2007) 2.79
Effective gene therapy with nonintegrating lentiviral vectors. Nat Med (2006) 2.70
Multiple vitamin K-dependent coagulation zymogens promote adenovirus-mediated gene delivery to hepatocytes. Blood (2006) 2.67
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther (2009) 2.56
Identification of coagulation factor (F)X binding sites on the adenovirus serotype 5 hexon: effect of mutagenesis on FX interactions and gene transfer. Blood (2009) 2.55
Exogenous control of mammalian gene expression through modulation of RNA self-cleavage. Nature (2004) 2.48
The Wiskott-Aldrich syndrome. J Allergy Clin Immunol (2006) 2.29
Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial. Hum Gene Ther (2011) 2.29
Clinical features and outcome of patients with IRAK-4 and MyD88 deficiency. Medicine (Baltimore) (2010) 2.26
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
The Biomedical Resource Ontology (BRO) to enable resource discovery in clinical and translational research. J Biomed Inform (2010) 2.10
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther (2006) 2.08
Crystal structure of human carboxylesterase 1 complexed with the Alzheimer's drug tacrine: from binding promiscuity to selective inhibition. Chem Biol (2003) 2.05
Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol Ther (2002) 2.03
Efficiency of transduction of highly purified murine hematopoietic stem cells by lentiviral and oncoretroviral vectors under conditions of minimal in vitro manipulation. Mol Ther (2005) 1.99
The CD46-Jagged1 interaction is critical for human TH1 immunity. Nat Immunol (2012) 1.97
Initial testing (stage 1) of the proteasome inhibitor bortezomib by the pediatric preclinical testing program. Pediatr Blood Cancer (2008) 1.95
Activated protein C therapy slows ALS-like disease in mice by transcriptionally inhibiting SOD1 in motor neurons and microglia cells. J Clin Invest (2009) 1.86
Improved response in high-risk neuroblastoma with protracted topotecan administration using a pharmacokinetically guided dosing approach. J Clin Oncol (2005) 1.85
Molecular characterization of the pediatric preclinical testing panel. Clin Cancer Res (2008) 1.84
Bleeding due to disruption of a cargo-specific ER-to-Golgi transport complex. Nat Genet (2003) 1.82
Stable gene transfer to muscle using non-integrating lentiviral vectors. Mol Ther (2007) 1.81
Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med (2011) 1.81
Ontogeny of CD24 in the human kidney. Kidney Int (2010) 1.81
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Sci Transl Med (2011) 1.81
Two novel activating mutations in the Wiskott-Aldrich syndrome protein result in congenital neutropenia. Blood (2006) 1.80
The cytoplasm of living cells behaves as a poroelastic material. Nat Mater (2013) 1.80
Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods. Hum Gene Ther Methods (2012) 1.79
The insulin-like growth factor-1 receptor-targeting antibody, CP-751,871, suppresses tumor-derived VEGF and synergizes with rapamycin in models of childhood sarcoma. Cancer Res (2009) 1.79
Globin lentiviral vector insertions can perturb the expression of endogenous genes in beta-thalassemic hematopoietic cells. Mol Ther (2008) 1.78
Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors. Hum Mol Genet (2009) 1.76
Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood (2003) 1.75
Angiogenesis inhibitors in a murine neuroblastoma model: quantitative assessment of intratumoral blood flow with contrast-enhanced gray-scale US. Radiology (2006) 1.75
Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection. Blood (2009) 1.73
The leukocyte podosome. Eur J Cell Biol (2005) 1.72
Initial testing (stage 1) of a monoclonal antibody (SCH 717454) against the IGF-1 receptor by the pediatric preclinical testing program. Pediatr Blood Cancer (2008) 1.71