PubRank

Seng H Cheng

Author PubWeight™ 126.53‹?›

Top papers

Rank Title Journal Year PubWeight™‹?›
1 Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron 2012 4.48
2 Targeting nuclear RNA for in vivo correction of myotonic dystrophy. Nature 2012 2.63
3 CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy. J Clin Invest 2010 2.62
4 Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Sci Transl Med 2011 2.56
5 Acid β-glucosidase mutants linked to Gaucher disease, Parkinson disease, and Lewy body dementia alter α-synuclein processing. Ann Neurol 2011 2.11
6 Hypoxia up-regulates expression of peroxisome proliferator-activated receptor gamma angiopoietin-related gene (PGAR) in cardiomyocytes: role of hypoxia inducible factor 1alpha. J Mol Cell Cardiol 2002 2.02
7 Hypoxia-inducible factor-1 mediates activation of cultured vascular endothelial cells by inducing multiple angiogenic factors. Circ Res 2003 2.00
8 CNS expression of glucocerebrosidase corrects alpha-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy. Proc Natl Acad Sci U S A 2011 1.90
9 Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis. Mol Ther 2008 1.82
10 Inhibiting glycosphingolipid synthesis improves glycemic control and insulin sensitivity in animal models of type 2 diabetes. Diabetes 2007 1.82
11 CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression. Nat Biotechnol 2008 1.73
12 Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy. Mol Ther 2010 1.63
13 A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol Genet Metab 2007 1.61
14 Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity. Mol Ther 2008 1.57
15 AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice. Mol Ther 2004 1.55
16 Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A. Mol Ther 2006 1.46
17 Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies. Proc Natl Acad Sci U S A 2013 1.45
18 Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid-DNA complexes to ovine lung segments. Mol Ther 2003 1.43
19 Adenovirus-mediated expression of beta-adrenergic receptor kinase C-terminus reduces intimal hyperplasia and luminal stenosis of arteriovenous polytetrafluoroethylene grafts in pigs. Circulation 2005 1.40
20 Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease. Mol Ther 2009 1.38
21 CpG-depleted plasmid DNA vectors with enhanced safety and long-term gene expression in vivo. Mol Ther 2002 1.37
22 Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. J Neurosci 2006 1.33
23 Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse. Proc Natl Acad Sci U S A 2007 1.27
24 Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice. Hum Gene Ther 2008 1.26
25 Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice. Biochem J 2005 1.24
26 AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice. Mol Ther 2010 1.22
27 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
28 Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice. J Neuropathol Exp Neurol 2008 1.19
29 Effective gene therapy in an authentic model of Tay-Sachs-related diseases. Proc Natl Acad Sci U S A 2006 1.18
30 Stabilization of vascular endothelial growth factor mRNA by hypoxia-inducible factor 1. Biochem Biophys Res Commun 2002 1.18
31 AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease. J Gene Med 2006 1.17
32 Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease. Mol Genet Metab 2010 1.17
33 Inhibiting glycosphingolipid synthesis ameliorates hepatic steatosis in obese mice. Hepatology 2009 1.16
34 Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice. Hum Mol Genet 2012 1.15
35 Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease. Proc Natl Acad Sci U S A 2005 1.14
36 Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease. Exp Neurol 2008 1.13
37 Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease. Mol Ther 2007 1.11
38 Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy. J Inherit Metab Dis 2010 1.10
39 Cell and gene-based therapies for the lysosomal storage diseases. Curr Gene Ther 2006 1.10
40 Inhibiting TGF-β activity improves respiratory function in mdx mice. Am J Pathol 2011 1.09
41 Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. Hum Gene Ther 2014 1.08
42 Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease. PLoS One 2010 1.04
43 Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo. Respir Res 2006 1.03
44 Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression. Mol Ther 2011 1.03
45 Hypoxia-inducible factor 1 mediates hypoxia-induced cardiomyocyte lipid accumulation by reducing the DNA binding activity of peroxisome proliferator-activated receptor alpha/retinoid X receptor. Biochem Biophys Res Commun 2007 1.01
46 Contribution of Toll-like receptor 9 signaling to the acute inflammatory response to nonviral vectors. Mol Ther 2004 1.00
47 Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice. J Biol Chem 2004 0.99
48 Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease. Hum Gene Ther 2002 0.98
49 Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis. Proc Natl Acad Sci U S A 2013 0.97
50 Gene transfer corrects acute GM2 gangliosidosis--potential therapeutic contribution of perivascular enzyme flow. Mol Ther 2012 0.97
51 Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy. Mol Ther 2013 0.97
52 AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease. Mol Ther 2005 0.97
53 Expression of constitutively stable hybrid hypoxia-inducible factor-1alpha protects cultured rat cardiomyocytes against simulated ischemia-reperfusion injury. Am J Physiol Cell Physiol 2004 0.97
54 Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease. Hum Gene Ther 2014 0.96
55 Increased hepatic insulin action in diet-induced obese mice following inhibition of glucosylceramide synthase. PLoS One 2010 0.95
56 Gene expression profiles in human cardiac cells subjected to hypoxia or expressing a hybrid form of HIF-1 alpha. Physiol Genomics 2002 0.94
57 Mutant GBA1 expression and synucleinopathy risk: first insights from cellular and mouse models. Neurodegener Dis 2012 0.94
58 Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease. PLoS One 2011 0.92
59 Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer. Biomaterials 2011 0.92
60 Systemic delivery of a Peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy. Nucleic Acid Ther 2013 0.92
61 Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model. Mol Ther 2002 0.91
62 Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain. Hum Mol Genet 2011 0.90
63 Lowering glycosphingolipid levels in CD4+ T cells attenuates T cell receptor signaling, cytokine production, and differentiation to the Th17 lineage. J Biol Chem 2011 0.90
64 Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease. PLoS One 2012 0.89
65 Reversibility of neuropathology in Tay-Sachs-related diseases. Hum Mol Genet 2013 0.89
66 Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice. Mol Genet Metab 2012 0.89
67 Ligation of cytotoxic T lymphocyte antigen-4 to T cell receptor inhibits T cell activation and directs differentiation into Foxp3+ regulatory T cells. J Biol Chem 2012 0.89
68 Differential effects of membrane and soluble Fas ligand on cardiomyocytes: role in ischemia/reperfusion injury. J Mol Cell Cardiol 2003 0.89
69 AAV8-mediated gene therapy prevents induced biochemical attacks of acute intermittent porphyria and improves neuromotor function. Mol Ther 2009 0.88
70 A hypoxic inducible factor-1 alpha hybrid enhances collateral development and reduces vascular leakage in diabetic rats. J Gene Med 2009 0.87
71 Prospects for the gene therapy of spinal muscular atrophy. Trends Mol Med 2011 0.87
72 Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies. Mol Ther 2012 0.87
73 Reducing the immunostimulatory activity of CpG-containing plasmid DNA vectors for non-viral gene therapy. Expert Opin Drug Deliv 2004 0.87
74 Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice. Mol Ther 2010 0.87
75 Rapid identification of novel functional promoters for gene therapy. J Mol Med (Berl) 2012 0.86
76 IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice. Neurobiol Dis 2011 0.86
77 The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep. Biomaterials 2013 0.86
78 Expression of angiopoietins in renal epithelial and clear cell carcinoma cells: regulation by hypoxia and participation in angiogenesis. Am J Physiol Renal Physiol 2004 0.86
79 Relationship between neuropathology and disease progression in the SOD1(G93A) ALS mouse. Exp Neurol 2010 0.85
80 Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung. J Gene Med 2007 0.85
81 Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease. Mol Ther 2002 0.84
82 Dexamethasone-mediated up-regulation of the mannose receptor improves the delivery of recombinant glucocerebrosidase to Gaucher macrophages. J Pharmacol Exp Ther 2003 0.84
83 A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis. Thorax 2013 0.84
84 Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A. Mol Ther 2012 0.84
85 Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease. PLoS One 2013 0.84
86 Adeno-associated virus-mediated expression of acid sphingomyelinase decreases atherosclerotic lesion formation in apolipoprotein E(-/-) mice. J Gene Med 2011 0.83
87 Myocardial expression of baculoviral p35 alleviates doxorubicin-induced cardiomyopathy in rats. Hum Gene Ther 2003 0.83
88 Identification of transfected cell types following non-viral gene transfer to the murine lung. J Gene Med 2007 0.83
89 Reducing glycosphingolipid biosynthesis in airway cells partially ameliorates disease manifestations in a mouse model of asthma. Int Immunol 2010 0.83
90 Disease progression in a mouse model of amyotrophic lateral sclerosis: the influence of chronic stress and corticosterone. FASEB J 2011 0.83
91 Optimizing aerosol gene delivery and expression in the ovine lung. Mol Ther 2007 0.82
92 Inhibition of osteoclastogenesis by prolyl hydroxylase inhibitor dimethyloxallyl glycine. J Bone Miner Metab 2010 0.82
93 DNA sequences in cationic lipid:pDNA-mediated systemic toxicities. Hum Gene Ther 2003 0.82
94 Adenovirus-mediated expression of p35 prevents hypoxia/reoxygenation injury by reducing reactive oxygen species and caspase activity. Cardiovasc Res 2002 0.81
95 The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways. Biomaterials 2009 0.81
96 Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease. Mol Genet Metab 2009 0.81
97 Limitations of the murine nose in the development of nonviral airway gene transfer. Am J Respir Cell Mol Biol 2009 0.81
98 Induction of immune tolerance to a therapeutic protein by intrathymic gene delivery. Mol Ther 2010 0.81
99 Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors. J Gene Med 2004 0.81
100 The chemosensitizing activity of inhibitors of glucosylceramide synthase is mediated primarily through modulation of P-gp function. Int J Oncol 2010 0.80
101 In vivo imaging of gene transfer to the respiratory tract. Biomaterials 2007 0.80
102 Low-frequency ultrasound increases non-viral gene transfer to the mouse lung. Acta Biochim Biophys Sin (Shanghai) 2010 0.79
103 Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouse. Exp Neurol 2007 0.79
104 Proteasome inhibition is partially effective in attenuating pre-existing immunity against recombinant adeno-associated viral vectors. PLoS One 2012 0.79
105 Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity. J Gene Med 2010 0.78
106 Acute intermittent porphyria: vector optimization for gene therapy. J Gene Med 2007 0.78
107 The role of doxorubicin in non-viral gene transfer in the lung. Biomaterials 2009 0.78
108 A novel mixing device for the reproducible generation of nonviral gene therapy formulations. Biotechniques 2010 0.77
109 Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases. Neurotherapeutics 2011 0.77
110 Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium. J Gene Med 2012 0.77
111 Fas ligand gene therapy for vascular intimal hyperplasia. Curr Gene Ther 2004 0.77
112 Erythrocytes encapsulated with phenylalanine hydroxylase exhibit improved pharmacokinetics and lowered plasma phenylalanine levels in normal mice. Mol Genet Metab 2013 0.77
113 Catheter-mediated delivery of adenoviral vectors expressing beta-adrenergic receptor kinase C-terminus inhibits intimal hyperplasia and luminal stenosis in rabbit iliac arteries. J Gene Med 2004 0.76
114 A bispecific protein capable of engaging CTLA-4 and MHCII protects non-obese diabetic mice from autoimmune diabetes. PLoS One 2013 0.75
115 Comparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular delivery. PLoS One 2011 0.75
116 Formulation of synthetic vectors for cystic fibrosis gene therapy. Methods Mol Med 2002 0.75
117 Formulation of synthetic gene delivery vectors for transduction of the airway epithelium. Methods Mol Biol 2004 0.75