Published in Blood on July 01, 2004
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
Rab38 and Rab32 control post-Golgi trafficking of melanogenic enzymes. J Cell Biol (2006) 2.79
Correction of ADAMTS13 deficiency by in utero gene transfer of lentiviral vector encoding ADAMTS13 genes. Mol Ther (2008) 1.05
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
Gene therapy for hemophilia. Front Biosci (Landmark Ed) (2015) 0.94
LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives. Int Arch Med (2010) 0.91
Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX. Gene Ther (2013) 0.91
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage. Blood (2011) 0.89
Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2. Mol Ther (2010) 0.86
Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance. Mol Ther (2015) 0.85
Perinatal gene transfer to the liver. Curr Pharm Des (2011) 0.84
Robust in vivo transduction of nervous system and neural stem cells by early gestational intra amniotic gene transfer using lentiviral vector. Mol Ther (2010) 0.84
The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis. Mol Ther (2012) 0.82
Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene. Gene Ther (2010) 0.80
Early fetal gene delivery utilizes both central and peripheral mechanisms of tolerance induction. Exp Hematol (2008) 0.80
Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector delivery. J Immunol Res (2015) 0.80
The fetal respiratory system as target for antenatal therapy. Facts Views Vis Obgyn (2011) 0.79
In utero therapy for congenital disorders using amniotic fluid stem cells. Front Pharmacol (2014) 0.79
The fetal patient -- ethical aspects of fetal therapy. Facts Views Vis Obgyn (2011) 0.78
A novel surgical approach for intratracheal administration of bioactive agents in a fetal mouse model. J Vis Exp (2012) 0.77
Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery. J Genet Syndr Gene Ther (2012) 0.77
In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application. Mol Ther Methods Clin Dev (2016) 0.76
Gene therapy to the kidney using viral vectors. Paidiatrike (2008) 0.75
Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep. PLoS One (2017) 0.75
In vivo gene transfer strategies to achieve partial correction of von Willebrand disease. Hum Gene Ther (2012) 0.75
Hemophilia A: an ideal disease to correct in utero. Front Pharmacol (2014) 0.75
Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes. Mol Ther Methods Clin Dev (2017) 0.75
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
Copy number polymorphism in Fcgr3 predisposes to glomerulonephritis in rats and humans. Nature (2006) 9.30
The Oxford classification of IgA nephropathy: rationale, clinicopathological correlations, and classification. Kidney Int (2009) 7.45
The Oxford classification of IgA nephropathy: pathology definitions, correlations, and reproducibility. Kidney Int (2009) 7.19
Mutations in VKORC1 cause warfarin resistance and multiple coagulation factor deficiency type 2. Nature (2004) 5.95
FCGR3B copy number variation is associated with susceptibility to systemic, but not organ-specific, autoimmunity. Nat Genet (2007) 5.93
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet (2004) 5.43
Adenovirus serotype 5 hexon mediates liver gene transfer. Cell (2008) 5.00
Uncontrolled C3 activation causes membranoproliferative glomerulonephritis in mice deficient in complement factor H. Nat Genet (2002) 4.22
Genetic loci influencing kidney function and chronic kidney disease. Nat Genet (2010) 3.75
Identification of a mutation in complement factor H-related protein 5 in patients of Cypriot origin with glomerulonephritis. Lancet (2010) 3.74
Pathologic classification of diabetic nephropathy. J Am Soc Nephrol (2010) 3.61
Membranoproliferative glomerulonephritis type II (dense deposit disease): an update. J Am Soc Nephrol (2005) 3.57
Prospective observational single-centre cohort study to evaluate the effectiveness of treating lupus nephritis with rituximab and mycophenolate mofetil but no oral steroids. Ann Rheum Dis (2013) 3.39
C3 glomerulopathy: a new classification. Nat Rev Nephrol (2010) 3.17
New approaches to the treatment of dense deposit disease. J Am Soc Nephrol (2007) 3.02
Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med (2014) 2.92
C3 glomerulopathy: consensus report. Kidney Int (2013) 2.85
A randomized controlled trial of peripheral blood mononuclear cell depletion in experimental human lung inflammation. Am J Respir Crit Care Med (2013) 2.71
Effective gene therapy with nonintegrating lentiviral vectors. Nat Med (2006) 2.70
Long-term effect of gene therapy on Leber's congenital amaurosis. N Engl J Med (2015) 2.68
Multiple vitamin K-dependent coagulation zymogens promote adenovirus-mediated gene delivery to hepatocytes. Blood (2006) 2.67
The future of gene therapy. Nature (2004) 2.65
Spontaneous hemolytic uremic syndrome triggered by complement factor H lacking surface recognition domains. J Exp Med (2007) 2.55
The bone marrow functionally contributes to liver fibrosis. Gastroenterology (2006) 2.52
Immunostaining findings in IgA nephropathy: correlation with histology and clinical outcome in the Oxford classification patient cohort. Nephrol Dial Transplant (2011) 2.51
Nr4a1-dependent Ly6C(low) monocytes monitor endothelial cells and orchestrate their disposal. Cell (2013) 2.49
The inhibiting Fc receptor for IgG, FcγRIIB, is a modifier of autoimmune susceptibility. J Immunol (2011) 2.44
Characterization and clinical application of human CD34+ stem/progenitor cell populations mobilized into the blood by granulocyte colony-stimulating factor. Stem Cells (2006) 2.30
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
Ccl2, Cx3cr1 and Ccl2/Cx3cr1 chemokine deficiencies are not sufficient to cause age-related retinal degeneration. Exp Eye Res (2012) 2.18
The Oxford IgA nephropathy clinicopathological classification is valid for children as well as adults. Kidney Int (2010) 2.14
Multiple organ engraftment by bone-marrow-derived myofibroblasts and fibroblasts in bone-marrow-transplanted mice. Stem Cells (2003) 2.03
Prolonged disease-free remission following rituximab and low-dose cyclophosphamide therapy for renal ANCA-associated vasculitis. Nephrol Dial Transplant (2011) 2.03
Retinal ganglion cell apoptosis in glaucoma is related to intraocular pressure and IOP-induced effects on extracellular matrix. Invest Ophthalmol Vis Sci (2005) 2.02
Activin A and TGF-β promote T(H)9 cell-mediated pulmonary allergic pathology. J Allergy Clin Immunol (2012) 1.98
Factor I is required for the development of membranoproliferative glomerulonephritis in factor H-deficient mice. J Clin Invest (2008) 1.94
Bone marrow-derived cells contribute to podocyte regeneration and amelioration of renal disease in a mouse model of Alport syndrome. Stem Cells (2006) 1.90
The development of atypical hemolytic uremic syndrome depends on complement C5. J Am Soc Nephrol (2010) 1.88
C1q deficiency and autoimmunity: the effects of genetic background on disease expression. J Immunol (2002) 1.88
Antineutrophil cytoplasm antibodies directed against myeloperoxidase augment leukocyte-microvascular interactions in vivo. Blood (2005) 1.86
The drusenlike phenotype in aging Ccl2-knockout mice is caused by an accelerated accumulation of swollen autofluorescent subretinal macrophages. Invest Ophthalmol Vis Sci (2009) 1.84
Repair of the degenerate retina by photoreceptor transplantation. Proc Natl Acad Sci U S A (2012) 1.79
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
Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood (2010) 1.68
Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of leber congenital amaurosis caused by guanylate cyclase-1 deficiency. Hum Gene Ther (2011) 1.68
Spontaneous autoimmunity in 129 and C57BL/6 mice-implications for autoimmunity described in gene-targeted mice. PLoS Biol (2004) 1.68
Inhibition of hypoxia inducible factor hydroxylases protects against renal ischemia-reperfusion injury. J Am Soc Nephrol (2008) 1.68
Familial C3 glomerulopathy associated with CFHR5 mutations: clinical characteristics of 91 patients in 16 pedigrees. Clin J Am Soc Nephrol (2011) 1.66
Regulation of PCNA and cyclin D1 expression and epithelial morphogenesis by the ZO-1-regulated transcription factor ZONAB/DbpA. Mol Cell Biol (2006) 1.65
Interferon-beta reduces proteinuria in experimental glomerulonephritis. J Am Soc Nephrol (2007) 1.63
Continuous high-titer HIV-1 vector production. Nat Biotechnol (2003) 1.58
Pharmacodynamic resistance to warfarin associated with a Val66Met substitution in vitamin K epoxide reductase complex subunit 1. Thromb Haemost (2005) 1.54
CD59a deficiency exacerbates accelerated nephrotoxic nephritis in mice. J Am Soc Nephrol (2003) 1.54
Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy. J Pediatr (2009) 1.52
Defining the integration capacity of embryonic stem cell-derived photoreceptor precursors. Stem Cells (2012) 1.52
Elimination of human leukemia cells in NOD/SCID mice by WT1-TCR gene-transduced human T cells. Blood (2005) 1.51
Elevated soluble Flt1 inhibits endothelial repair in PR3-ANCA-associated vasculitis. J Am Soc Nephrol (2011) 1.50
ABO incompatible living renal transplantation with a steroid sparing protocol. Transplantation (2008) 1.48
Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy. Hum Mol Genet (2011) 1.47
Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP. Invest Ophthalmol Vis Sci (2005) 1.47
Bone marrow-derived cells do not contribute significantly to collagen I synthesis in a murine model of renal fibrosis. J Am Soc Nephrol (2006) 1.46
Autologous transplantation of the retinal pigment epithelium and choroid in the treatment of neovascular age-related macular degeneration. Ophthalmology (2007) 1.44
AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter. Invest Ophthalmol Vis Sci (2007) 1.44
Long-term survival of photoreceptors transplanted into the adult murine neural retina requires immune modulation. Stem Cells (2010) 1.42
Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease. PLoS One (2010) 1.42
Proliferation of bone marrow-derived cells contributes to regeneration after folic acid-induced acute tubular injury. J Am Soc Nephrol (2005) 1.41
Non-hepatitis virus associated mixed essential cryoglobulinemia. Kidney Int (2009) 1.40
Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther (2005) 1.38
Autosomal dominant erythrocytosis and pulmonary arterial hypertension associated with an activating HIF2 alpha mutation. Blood (2008) 1.37
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood (2013) 1.36
Genetic dissection of spontaneous autoimmunity driven by 129-derived chromosome 1 Loci when expressed on C57BL/6 mice. J Immunol (2007) 1.34
Jund is a determinant of macrophage activation and is associated with glomerulonephritis susceptibility. Nat Genet (2008) 1.31
Targeting of adenovirus serotype 5 (Ad5) and 5/47 pseudotyped vectors in vivo: fundamental involvement of coagulation factors and redundancy of CAR binding by Ad5. J Virol (2007) 1.29
AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa. Mol Ther (2003) 1.26
Role of intestinal subepithelial myofibroblasts in inflammation and regenerative response in the gut. Pharmacol Ther (2007) 1.26
Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice. Mol Genet Metab (2009) 1.26
Biodistribution and retargeting of FX-binding ablated adenovirus serotype 5 vectors. Blood (2010) 1.25
Treatment with human complement factor H rapidly reverses renal complement deposition in factor H-deficient mice. Kidney Int (2010) 1.25
HIF-1alpha and HIF-2alpha are differentially activated in distinct cell populations in retinal ischaemia. PLoS One (2010) 1.24
Analytical strategies for characterization of oxysterol lipidomes: liver X receptor ligands in plasma. Free Radic Biol Med (2012) 1.23
The epidemiology, diagnosis, and management of aristolochic acid nephropathy: a narrative review. Ann Intern Med (2013) 1.23
Topographical characterization of cone photoreceptors and the area centralis of the canine retina. Mol Vis (2008) 1.23
Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system. FASEB J (2011) 1.22
Experimental autoimmune vasculitis: an animal model of anti-neutrophil cytoplasmic autoantibody-associated systemic vasculitis. Am J Pathol (2009) 1.21
Recent insights into C3 glomerulopathy. Nephrol Dial Transplant (2013) 1.21
Prospects for retinal gene replacement therapy. Trends Genet (2009) 1.20
Differential modulation of retinal degeneration by Ccl2 and Cx3cr1 chemokine signalling. PLoS One (2012) 1.19
C3 glomerulopathy: clinicopathologic features and predictors of outcome. Clin J Am Soc Nephrol (2013) 1.19