Published in Mol Ther on June 07, 2013
Clinical applications involving CNS gene transfer. Adv Genet (2014) 0.89
Methods for gene transfer to the central nervous system. Adv Genet (2014) 0.88
Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10year combined experience of 2 centres. Mol Genet Metab (2016) 0.88
Gene therapy for the neurological manifestations in lysosomal storage disorders. J Lipid Res (2014) 0.83
Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery. Mol Ther Methods Clin Dev (2016) 0.82
Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I. J Biol Chem (2014) 0.82
Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice. Mol Ther (2015) 0.81
Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome. Orphanet J Rare Dis (2015) 0.80
Quantitative assessment of barriers to the clinical development and adoption of cellular therapies: A pilot study. J Tissue Eng (2014) 0.80
Gene therapy for neurologic manifestations of mucopolysaccharidoses. Expert Opin Drug Deliv (2014) 0.79
Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase. Mol Ther (2014) 0.77
Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier. PLoS One (2013) 0.77
Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier. Mol Genet Metab (2014) 0.76
Checkpoints to the Brain: Directing Myeloid Cell Migration to the Central Nervous System. Int J Mol Sci (2016) 0.75
Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications. Mol Ther (2017) 0.75
Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB. Metab Brain Dis (2017) 0.75
A third-generation lentivirus vector with a conditional packaging system. J Virol (1998) 16.07
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science (2009) 8.80
A gene network regulating lysosomal biogenesis and function. Science (2009) 7.17
DAMPening inflammation by modulating TLR signalling. Mediators Inflamm (2010) 3.29
Sanfilippo syndrome: a mini-review. J Inherit Metab Dis (2008) 2.35
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med (2010) 1.84
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
Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders. J Comp Neurol (2004) 1.74
Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant (2007) 1.72
A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome). Glycobiology (1999) 1.67
Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J Inherit Metab Dis (1995) 1.62
Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest (2006) 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
Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases. Cell Transplant (1995) 1.42
Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease. PLoS One (2010) 1.42
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood (2010) 1.33
Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome. Biol Blood Marrow Transplant (2009) 1.32
Scoring evaluation of the natural course of mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A). Pediatrics (2007) 1.31
Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice. PLoS One (2008) 1.30
Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece. Am J Med Genet A (2011) 1.26
Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes. Hum Mol Genet (2007) 1.20
Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer. Hum Gene Ther (2012) 1.14
Identification of the promoter of the myelomonocytic leukocyte integrin CD11b. Proc Natl Acad Sci U S A (1992) 1.13
Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation. Proc Natl Acad Sci U S A (2012) 1.12
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
Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans. J Inherit Metab Dis (2012) 1.09
Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB. PLoS One (2012) 1.08
Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS. Gene Ther (2009) 1.08
Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA. Mol Genet Metab (2004) 1.04
Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA. Exp Neurol (2011) 1.04
Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases. J Inherit Metab Dis (2010) 1.01
Identification and sequence analysis of the promoter for the leukocyte integrin beta-subunit (CD18): a retinoic acid-inducible gene. Blood (1992) 1.01
Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice. Mol Ther (2012) 1.00
Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB. Behav Brain Res (2010) 0.99
Heparan sulfate and dermatan sulfate derived disaccharides are sensitive markers for newborn screening for mucopolysaccharidoses types I, II and III. Mol Genet Metab (2012) 0.99
Evaluation of heparin cofactor II-thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice. Mol Genet Metab (2009) 0.97
Busulfan conditioning enhances engraftment of hematopoietic donor-derived cells in the brain compared with irradiation. Mol Ther (2013) 0.97
Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test. PLoS One (2011) 0.93
Mucopolysaccharidosis type I, unique structure of accumulated heparan sulfate and increased N-sulfotransferase activity in mice lacking α-l-iduronidase. J Biol Chem (2011) 0.93
Hyperactive behaviour in the mouse model of mucopolysaccharidosis IIIB in the open field and home cage environments. Genes Brain Behav (2011) 0.93
Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells. Mol Ther (2005) 0.91
Bone marrow transplantation in mucopolysaccharidosis type IIIA: a comparison of an early treated patient with his untreated sibling. J Inherit Metab Dis (1999) 0.89
Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity. J Inherit Metab Dis (2013) 0.89
Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy. Haematologica (2012) 0.88
Neuroinflammatory and oxidative stress phenomena in MPS IIIA mouse model: the positive effect of long-term aspirin treatment. Mol Genet Metab (2011) 0.86
Retroviral-mediated gene expression in human myelomonocytic cells: a comparison of hematopoietic cell promoters to viral promoters. Blood (1995) 0.77
Long-term neuropsychological follow-up of a child with Klüver-Bucy syndrome. Epilepsy Behav (2010) 0.76
Interleukin-6 biology is coordinated by membrane-bound and soluble receptors: role in inflammation and cancer. J Leukoc Biol (2006) 3.45
Study of telomere length reveals rapid aging of human marrow stromal cells following in vitro expansion. Stem Cells (2004) 3.15
The bone marrow functionally contributes to liver fibrosis. Gastroenterology (2006) 2.52
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics (2009) 2.44
Calcification is associated with loss of functional calcium-sensing receptor in vascular smooth muscle cells. Cardiovasc Res (2008) 2.23
The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I. Mol Genet Metab (2007) 2.23
Epithelial-mesenchymal transition events during human embryonic stem cell differentiation. Cancer Res (2007) 2.10
Hyperactivation of Stat3 in gp130 mutant mice promotes gastric hyperproliferation and desensitizes TGF-beta signaling. Nat Med (2005) 2.06
Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease. Genet Med (2012) 2.05
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab (2008) 2.04
Regional deposition of mometasone furoate nasal spray suspension in humans. Allergy Asthma Proc (2015) 2.04
Hemophagocytosis by leukemic megakaryoblasts in acute myeloid leukemia (megakaryoblastic) with t(1;22)(p13;q13);RBM15-MKL1. J Pediatr Hematol Oncol (2012) 2.00
Apoptosis is a natural stimulus of IL6R shedding and contributes to the proinflammatory trans-signaling function of neutrophils. Blood (2007) 1.92
The role of human HtrA1 in arthritic disease. J Biol Chem (2005) 1.91
Interleukin 6 (IL-6) deficiency delays lupus nephritis in MRL-Faslpr mice: the IL-6 pathway as a new therapeutic target in treatment of autoimmune kidney disease in systemic lupus erythematosus. J Rheumatol (2009) 1.84
Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease. Hepatology (2013) 1.78
Zoonosis emergence linked to agricultural intensification and environmental change. Proc Natl Acad Sci U S A (2013) 1.74
A quantifiable proliferative burst of tissue macrophages restores homeostatic macrophage populations after acute inflammation. Eur J Immunol (2011) 1.70
Human complete Stat-1 deficiency is associated with defective type I and II IFN responses in vitro but immunity to some low virulence viruses in vivo. J Immunol (2006) 1.69
Interplay between IFN-gamma and IL-6 signaling governs neutrophil trafficking and apoptosis during acute inflammation. J Clin Invest (2003) 1.69
IL-6 regulates neutrophil trafficking during acute inflammation via STAT3. J Immunol (2008) 1.69
Soluble IL-6 receptor governs IL-6 activity in experimental arthritis: blockade of arthritis severity by soluble glycoprotein 130. J Immunol (2003) 1.68
IL-6 trans-signaling via STAT3 directs T cell infiltration in acute inflammation. Proc Natl Acad Sci U S A (2005) 1.66
Abrogation of E-cadherin-mediated cell-cell contact in mouse embryonic stem cells results in reversible LIF-independent self-renewal. Stem Cells (2009) 1.63
Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis (2014) 1.60
Distinct bone marrow-derived and tissue-resident macrophage lineages proliferate at key stages during inflammation. Nat Commun (2013) 1.58
The heparanome--the enigma of encoding and decoding heparan sulfate sulfation. J Biotechnol (2007) 1.57
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure. Orphanet J Rare Dis (2011) 1.54
Circulating neutrophils maintain physiological blood pressure by suppressing bacteria and IFNgamma-dependent iNOS expression in the vasculature of healthy mice. Blood (2008) 1.53
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
The impact of monitoring Epstein-Barr virus PCR in paediatric bone marrow transplant patients: can it successfully predict outcome and guide intervention? Pediatr Blood Cancer (2006) 1.49
Heparan sulfate 6-O-endosulfatases: discrete in vivo activities and functional co-operativity. Biochem J (2006) 1.48
Implications of the serine protease HtrA1 in amyloid precursor protein processing. Proc Natl Acad Sci U S A (2005) 1.47
A role for IL-27p28 as an antagonist of gp130-mediated signaling. Nat Immunol (2010) 1.44
Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease. PLoS One (2010) 1.42
A new model for the domain structure of heparan sulfate based on the novel specificity of K5 lyase. J Biol Chem (2004) 1.42
Spatial analysis of sleeping sickness, southeastern Uganda, 1970-2003. Emerg Infect Dis (2006) 1.39
12/15-Lipoxygenase regulates the inflammatory response to bacterial products in vivo. J Immunol (2008) 1.38
BCSH/BSBMT guideline: diagnosis and management of veno-occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation. Br J Haematol (2013) 1.38
Transgenic blockade of interleukin 6 transsignaling abrogates inflammation. Blood (2007) 1.37
Loss of CD4+ T cell IL-6R expression during inflammation underlines a role for IL-6 trans signaling in the local maintenance of Th17 cells. J Immunol (2010) 1.36
Regulation of pre-B cell colony-enhancing factor by STAT-3-dependent interleukin-6 trans-signaling: implications in the pathogenesis of rheumatoid arthritis. Arthritis Rheum (2006) 1.36
Therapeutic targeting of IL-6 trans signaling counteracts STAT3 control of experimental inflammatory arthritis. J Immunol (2009) 1.35
Exercise in pregnancy and the postpartum period. J Obstet Gynaecol Can (2003) 1.33
Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Blood (2004) 1.30
Inhibition of classic signaling is a novel function of soluble glycoprotein 130 (sgp130), which is controlled by the ratio of interleukin 6 and soluble interleukin 6 receptor. J Biol Chem (2011) 1.29
E-cadherin inhibits cell surface localization of the pro-migratory 5T4 oncofetal antigen in mouse embryonic stem cells. Mol Biol Cell (2007) 1.26
Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice. Mol Genet Metab (2009) 1.26
The problem of choice: current biologic agents and future prospects in RA. Nat Rev Rheumatol (2013) 1.24
Analytical strategies for characterization of oxysterol lipidomes: liver X receptor ligands in plasma. Free Radic Biol Med (2012) 1.23
Essential alterations of heparan sulfate during the differentiation of embryonic stem cells to Sox1-enhanced green fluorescent protein-expressing neural progenitor cells. Stem Cells (2007) 1.23
Soluble TLR2 reduces inflammation without compromising bacterial clearance by disrupting TLR2 triggering. J Immunol (2009) 1.22
Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders. Pediatr Clin North Am (2010) 1.21
Interleukin-6 is crucial for recall of influenza-specific memory CD4 T cells. PLoS Pathog (2008) 1.21
Phosphatidylethanolamine-esterified eicosanoids in the mouse: tissue localization and inflammation-dependent formation in Th-2 disease. J Biol Chem (2009) 1.18
Using remote sensing and geographic information systems to identify villages at high risk for rhodesiense sleeping sickness in Uganda. Trans R Soc Trop Med Hyg (2005) 1.18
Contrast-induced acute kidney injury: specialty-specific protocols for interventional radiology, diagnostic computed tomography radiology, and interventional cardiology. Mayo Clin Proc (2009) 1.16
Differential regulation of neutrophil-activating chemokines by IL-6 and its soluble receptor isoforms. J Immunol (2004) 1.14
Characterization of surgical procedures in patients with mucopolysaccharidosis type I: findings from the MPS I Registry. J Pediatr (2009) 1.13
Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. J Pediatr Rehabil Med (2010) 1.11
Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans. J Inherit Metab Dis (2012) 1.09
Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB. PLoS One (2012) 1.08
Functional characterization of a soluble gp130 isoform and its therapeutic capacity in an experimental model of inflammatory arthritis. Arthritis Rheum (2006) 1.07
Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. J Pediatr (2009) 1.07
Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. J Inherit Metab Dis (2010) 1.06
Dexamethasone downregulates calcification-inhibitor molecules and accelerates osteogenic differentiation of vascular pericytes: implications for vascular calcification. Circ Res (2006) 1.05
Sleep enhances IL-6 trans-signaling in humans. FASEB J (2006) 1.03
A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD). Blood (2013) 1.03
Specific glycosaminoglycans modulate neural specification of mouse embryonic stem cells. Stem Cells (2011) 1.03
Esterified eicosanoids are acutely generated by 5-lipoxygenase in primary human neutrophils and in human and murine infection. Blood (2010) 1.02
Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II. J Inherit Metab Dis (2010) 1.02
Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases. J Inherit Metab Dis (2010) 1.01
Differential dependencies of monocytes and neutrophils on dectin-1, dectin-2 and complement for the recognition of fungal particles in inflammation. PLoS One (2012) 1.01
Macrophage heterogeneity and acute inflammation. Eur J Immunol (2011) 1.01
Naive and activated T cells display differential responsiveness to TL1A that affects Th17 generation, maintenance, and proliferation. FASEB J (2010) 1.01
Enhanced neutrophil migratory activity following major blunt trauma. Injury (2005) 1.01
Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice. Mol Ther (2012) 1.00
Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): data from the Hunter Outcome Survey. Genet Med (2010) 0.99
Cattle owners' perceptions of African bovine trypanosomiasis and its control in Busia and Kwale Districts of Kenya. Acta Trop (2003) 0.99
Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB. Behav Brain Res (2010) 0.99
Sleeping sickness in Uganda: revisiting current and historical distributions. Afr Health Sci (2006) 0.99
Foxp3⁺ Treg cells in the inflamed CNS are insensitive to IL-6-driven IL-17 production. Eur J Immunol (2012) 0.98
Interferon-gamma protects against the development of structural damage in experimental arthritis by regulating polymorphonuclear neutrophil influx into diseased joints. Arthritis Rheum (2007) 0.98
Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A (2014) 0.98
The response of interleukin-6 and soluble interleukin-6 receptor isoforms following intermittent high intensity and continuous moderate intensity cycling. Cell Stress Chaperones (2010) 0.97