Published in Science on July 27, 2007
Genomic and epigenetic alterations deregulate microRNA expression in human epithelial ovarian cancer. Proc Natl Acad Sci U S A (2008) 4.79
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
Assessing the potential for AAV vector genotoxicity in a murine model. Blood (2010) 2.48
A largely random AAV integration profile after LPLD gene therapy. Nat Med (2013) 2.42
A transposon and transposase system for human application. Mol Ther (2010) 2.28
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood (2013) 2.09
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy. Blood (2008) 1.98
Human studies of angiogenic gene therapy. Circ Res (2009) 1.92
Mesenchymal stem cells for the treatment of neurodegenerative disease. Regen Med (2010) 1.89
Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas. Nat Genet (2015) 1.86
A modified sleeping beauty transposon system that can be used to model a wide variety of human cancers in mice. Cancer Res (2009) 1.86
Gene therapy in heart failure. Circ Res (2008) 1.79
Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection. Nat Protoc (2007) 1.73
SnoRNA U50 is a candidate tumor-suppressor gene at 6q14.3 with a mutation associated with clinically significant prostate cancer. Hum Mol Genet (2008) 1.62
Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy. Hepatology (2012) 1.59
Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature (2014) 1.57
Small nucleolar RNA signatures as biomarkers for non-small-cell lung cancer. Mol Cancer (2010) 1.56
Small nucleolar RNA 42 acts as an oncogene in lung tumorigenesis. Oncogene (2011) 1.54
Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. J Clin Invest (2015) 1.46
Recombinant adeno-associated virus transduction and integration. Mol Ther (2008) 1.44
Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle. J Virol (2008) 1.44
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
Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation. Nucleic Acids Res (2010) 1.42
Genetic enhancement of stem cell engraftment, survival, and efficacy. Circ Res (2008) 1.42
Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology (2010) 1.41
Induction of hepatocellular carcinoma by in vivo gene targeting. Proc Natl Acad Sci U S A (2012) 1.31
Immunologic privilege in the central nervous system and the blood-brain barrier. J Cereb Blood Flow Metab (2012) 1.30
Gene therapy for mucopolysaccharidosis. Expert Opin Biol Ther (2007) 1.28
Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? Hum Gene Ther (2015) 1.25
Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol Ther (2010) 1.21
Adeno-associated virus vector integration. Curr Opin Mol Ther (2009) 1.20
Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity. Mol Ther (2012) 1.19
Ku70, an essential gene, modulates the frequency of rAAV-mediated gene targeting in human somatic cells. Proc Natl Acad Sci U S A (2008) 1.19
Identification of rtl1, a retrotransposon-derived imprinted gene, as a novel driver of hepatocarcinogenesis. PLoS Genet (2013) 1.15
Small nucleolar RNAs in cancer. Biochim Biophys Acta (2012) 1.11
The gene therapy journey for hemophilia: are we there yet? Blood (2012) 1.10
Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs. Gene Ther (2012) 1.09
Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer. Nat Methods (2013) 1.08
Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy. Genome Biol (2007) 1.08
Advances in microRNAs: implications for gene therapists. Hum Gene Ther (2008) 1.08
Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies. Curr Gene Ther (2011) 1.08
Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Mol Ther (2012) 1.08
MicroRNA-494 within an oncogenic microRNA megacluster regulates G1/S transition in liver tumorigenesis through suppression of mutated in colorectal cancer. Hepatology (2013) 1.07
Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis. Ann Neurol (2012) 1.05
Cell- and gene-based therapeutic strategies for periodontal regenerative medicine. J Periodontol (2011) 1.04
Animal models of hemophilia. Prog Mol Biol Transl Sci (2012) 1.04
Crossing the rubicon. Nat Biotechnol (2009) 1.03
Lentiviral gene transfer into the dorsal root ganglion of adult rats. Mol Pain (2011) 1.03
Mechanisms of nucleotide trafficking during siRNA delivery to endothelial cells using perfluorocarbon nanoemulsions. Biomaterials (2010) 1.02
CNS-directed gene therapy for lysosomal storage diseases. Acta Paediatr Suppl (2008) 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
Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver. Hepatology (2014) 1.00
Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver. Mol Ther (2012) 0.98
Group II intron-based gene targeting reactions in eukaryotes. PLoS One (2008) 0.98
Adeno-associated Vector Toxicity-To Be or Not to Be? Mol Ther (2015) 0.98
High-throughput sequencing reveals principles of adeno-associated virus serotype 2 integration. J Virol (2013) 0.97
Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery. PLoS One (2012) 0.96
Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs. Hum Gene Ther Clin Dev (2015) 0.95
Oncolytic virotherapy for malignant glioma: translating laboratory insights into clinical practice. Front Oncol (2013) 0.95
Differential effects of DNA double-strand break repair pathways on single-strand and self-complementary adeno-associated virus vector genomes. J Virol (2010) 0.95
Frequency and spectrum of genomic integration of recombinant adeno-associated virus serotype 8 vector in neonatal mouse liver. J Virol (2008) 0.94
Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons. Hum Gene Ther (2010) 0.92
Retroviral vectors: post entry events and genomic alterations. Viruses (2011) 0.92
Transfection of cells using flow-through electroporation based on constant voltage. Nat Protoc (2011) 0.92
Displaying high-affinity ligands on adeno-associated viral vectors enables tumor cell-specific and safe gene transfer. Mol Ther (2012) 0.92
Hepatocyte growth factor/Met gene transfer in cardiac stem cells--potential for cardiac repair. Basic Res Cardiol (2010) 0.92
Cell penetrating peptide POD mediates delivery of recombinant proteins to retina, cornea and skin. Vision Res (2009) 0.91
Gene therapy for the treatment of chronic peripheral nervous system pain. Neurobiol Dis (2012) 0.91
Adeno-associated virus-mediated gene transfer. J Cell Biochem (2008) 0.91
AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo. Hum Gene Ther (2012) 0.90
Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia. J Genet Syndr Gene Ther (2012) 0.90
Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates. Mol Ther (2014) 0.90
Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. Hum Gene Ther (2013) 0.88
Development of a hybrid baculoviral vector for sustained transgene expression. Mol Ther (2009) 0.88
Barriers for retinal gene therapy: separating fact from fiction. Vision Res (2008) 0.88
AAV-mediated liver-directed gene therapy. Methods Mol Biol (2011) 0.88
Adeno-associated virus finds its disease. Nat Genet (2015) 0.87
Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges. Expert Rev Hematol (2011) 0.87
Baculovirus transduction of mesenchymal stem cells: in vitro responses and in vivo immune responses after cell transplantation. Mol Ther (2009) 0.87
Epstein-Barr virus nuclear antigen 1 does not cause lymphoma in C57BL/6J mice. J Virol (2008) 0.87
Gene therapy targeting nuclear factor-kappaB: towards clinical application in inflammatory diseases and cancer. Curr Gene Ther (2009) 0.86
Viral vectors for in vivo gene transfer in Parkinson's disease: properties and clinical grade production. Exp Neurol (2007) 0.86
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice. Gene Ther (2013) 0.86
Gene correction in human embryonic and induced pluripotent stem cells: promises and challenges ahead. Mol Ther (2010) 0.86
Angiopoietin-1-expressing adipose stem cells genetically modified with baculovirus nanocomplex: investigation in rat heart with acute infarction. Int J Nanomedicine (2012) 0.86
Regulatory role of small nucleolar RNAs in human diseases. Biomed Res Int (2015) 0.85
Adeno-associated viral vectors for the treatment of hemophilia. Hum Mol Genet (2015) 0.85
Recombinant AAV-directed gene therapy for type I glycogen storage diseases. Expert Opin Biol Ther (2011) 0.85
Analysis of small nucleolar RNAs in sputum for lung cancer diagnosis. Oncotarget (2016) 0.84
Impact of hydrodynamic injection and phiC31 integrase on tumor latency in a mouse model of MYC-induced hepatocellular carcinoma. PLoS One (2010) 0.84
Perinatal gene transfer to the liver. Curr Pharm Des (2011) 0.84
Successful gene therapy in utero for lethal murine hypophosphatasia. Hum Gene Ther (2012) 0.84
AAV provides an alternative for gene therapy of the peripheral sensory nervous system. Mol Ther (2010) 0.84
Preferential targeting of disseminated liver tumors using a recombinant adeno-associated viral vector. Hum Gene Ther (2015) 0.83
Expression of non-coding RNA AB063319 derived from Rian gene during mouse development. J Mol Histol (2011) 0.83
Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications. Mol Ther (2015) 0.83
Regulation of snoRNAs in cancer: close encounters with interferon. J Interferon Cytokine Res (2013) 0.83
Progress toward cell-directed therapy for phenylketonuria. Clin Genet (2008) 0.83
Gene therapy for the neurological manifestations in lysosomal storage disorders. J Lipid Res (2014) 0.83
Design and validation of a histological scoring system for nonalcoholic fatty liver disease. Hepatology (2005) 29.57
Pioglitazone, vitamin E, or placebo for nonalcoholic steatohepatitis. N Engl J Med (2010) 13.33
A key role for autophagy and the autophagy gene Atg16l1 in mouse and human intestinal Paneth cells. Nature (2008) 10.84
Genomewide association study of leprosy. N Engl J Med (2009) 8.17
An intergroup phase III comparison of standard radiation therapy and two schedules of concurrent chemoradiotherapy in patients with unresectable squamous cell head and neck cancer. J Clin Oncol (2003) 6.82
A comprehensive classification system for lipids. J Lipid Res (2005) 6.52
Genome-wide association study in a Chinese Han population identifies nine new susceptibility loci for systemic lupus erythematosus. Nat Genet (2009) 6.42
LMSD: LIPID MAPS structure database. Nucleic Acids Res (2006) 6.21
Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med (2004) 5.59
The diagnosis and management of non-alcoholic fatty liver disease: practice guideline by the American Gastroenterological Association, American Association for the Study of Liver Diseases, and American College of Gastroenterology. Gastroenterology (2012) 5.47
Transplanted bone marrow regenerates liver by cell fusion. Nature (2003) 5.06
A unifying genetic model for facioscapulohumeral muscular dystrophy. Science (2010) 4.87
Intravenous administration of human bone marrow stromal cells induces angiogenesis in the ischemic boundary zone after stroke in rats. Circ Res (2003) 4.29
Lipidomics reveals a remarkable diversity of lipids in human plasma. J Lipid Res (2010) 4.14
US Renal Data System 2014 Annual Data Report: Epidemiology of Kidney Disease in the United States. Am J Kidney Dis (2015) 3.94
Clinical importance of the cytochromes P450. Lancet (2002) 3.77
Statins induce angiogenesis, neurogenesis, and synaptogenesis after stroke. Ann Neurol (2003) 3.54
Induction of ovarian cancer by defined multiple genetic changes in a mouse model system. Cancer Cell (2002) 3.50
Endothelial infection with KSHV genes in vivo reveals that vGPCR initiates Kaposi's sarcomagenesis and can promote the tumorigenic potential of viral latent genes. Cancer Cell (2003) 3.42
Effect of vitamin E or metformin for treatment of nonalcoholic fatty liver disease in children and adolescents: the TONIC randomized controlled trial. JAMA (2011) 3.37
Nonalcoholic fatty liver disease: pathology and pathogenesis. Annu Rev Pathol (2010) 3.36
Uniqueness theorems in bioluminescence tomography. Med Phys (2004) 3.32
Improved nonalcoholic steatohepatitis after 48 weeks of treatment with the PPAR-gamma ligand rosiglitazone. Hepatology (2003) 3.23
Progressive fibrosis in nonalcoholic steatohepatitis: association with altered regeneration and a ductular reaction. Gastroenterology (2007) 3.18
Nomenclature of the finer branches of the biliary tree: canals, ductules, and ductular reactions in human livers. Hepatology (2004) 3.00
Directed evolution of human T-cell receptors with picomolar affinities by phage display. Nat Biotechnol (2005) 2.97
Facioscapulohumeral dystrophy: incomplete suppression of a retrotransposed gene. PLoS Genet (2010) 2.95
The genetic map of Artemisia annua L. identifies loci affecting yield of the antimalarial drug artemisinin. Science (2010) 2.92
Genetic evidence that the human CYP2R1 enzyme is a key vitamin D 25-hydroxylase. Proc Natl Acad Sci U S A (2004) 2.89
Hepatocellular carcinoma in non-alcoholic fatty liver disease: an emerging menace. J Hepatol (2012) 2.89
Treatment of neural injury with marrow stromal cells. Lancet Neurol (2002) 2.89
Orlistat for overweight subjects with nonalcoholic steatohepatitis: A randomized, prospective trial. Hepatology (2009) 2.88
Clinical, laboratory and histological associations in adults with nonalcoholic fatty liver disease. Hepatology (2010) 2.86
Randomized phase III evaluation of cisplatin plus fluorouracil versus cisplatin plus paclitaxel in advanced head and neck cancer (E1395): an intergroup trial of the Eastern Cooperative Oncology Group. J Clin Oncol (2005) 2.83
Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2. Nat Genet (2012) 2.82
Targeted transgene insertion into human chromosomes by adeno-associated virus vectors. Nat Biotechnol (2002) 2.73
Loss of nuclear receptor SHP impairs but does not eliminate negative feedback regulation of bile acid synthesis. Dev Cell (2002) 2.68
Atorvastatin induction of VEGF and BDNF promotes brain plasticity after stroke in mice. J Cereb Blood Flow Metab (2005) 2.65
Control of HIV-1 immune escape by CD8 T cells expressing enhanced T-cell receptor. Nat Med (2008) 2.64
A genome-wide association study of nasopharyngeal carcinoma identifies three new susceptibility loci. Nat Genet (2010) 2.62
Intravenous bone marrow stromal cell therapy reduces apoptosis and promotes endogenous cell proliferation after stroke in female rat. J Neurosci Res (2003) 2.61
Amniotic infection syndrome: nosology and reproducibility of placental reaction patterns. Pediatr Dev Pathol (2004) 2.59
A suppressor screen in Mecp2 mutant mice implicates cholesterol metabolism in Rett syndrome. Nat Genet (2013) 2.59
Foamy virus vector integration sites in normal human cells. Proc Natl Acad Sci U S A (2006) 2.52
RNA transcripts, miRNA-sized fragments and proteins produced from D4Z4 units: new candidates for the pathophysiology of facioscapulohumeral dystrophy. Hum Mol Genet (2009) 2.51
Enzymatic reduction of oxysterols impairs LXR signaling in cultured cells and the livers of mice. Cell Metab (2007) 2.51
CD4-CD8 lineage commitment is regulated by a silencer element at the ThPOK transcription-factor locus. Immunity (2008) 2.49
Severe NAFLD with hepatic necroinflammatory changes in mice fed trans fats and a high-fructose corn syrup equivalent. Am J Physiol Gastrointest Liver Physiol (2008) 2.45
A genome-wide association study identifies novel and functionally related susceptibility Loci for Kawasaki disease. PLoS Genet (2009) 2.45
Endpoints and clinical trial design for nonalcoholic steatohepatitis. Hepatology (2011) 2.44
The use of liver biopsy evaluation in discrimination of idiopathic autoimmune hepatitis versus drug-induced liver injury. Hepatology (2011) 2.42
Ischemic rat brain extracts induce human marrow stromal cell growth factor production. Neuropathology (2002) 2.38
Regulated accumulation of desmosterol integrates macrophage lipid metabolism and inflammatory responses. Cell (2012) 2.38
Increased fibrillar amyloid-{beta} burden in normal individuals with a family history of late-onset Alzheimer's. Proc Natl Acad Sci U S A (2010) 2.32
Arabidopsis ABA INSENSITIVE4 regulates lipid mobilization in the embryo and reveals repression of seed germination by the endosperm. Plant Cell (2006) 2.27
Adeno-associated virus vectors integrate at chromosome breakage sites. Nat Genet (2004) 2.27
Principled sure independence screening for Cox models with ultra-high-dimensional covariates. J Multivar Anal (2012) 2.23
Hypometabolism exceeds atrophy in presymptomatic early-onset familial Alzheimer's disease. J Nucl Med (2006) 2.17
Physical activity recommendations, exercise intensity, and histological severity of nonalcoholic fatty liver disease. Am J Gastroenterol (2011) 2.15
FDG-PET changes in brain glucose metabolism from normal cognition to pathologically verified Alzheimer's disease. Eur J Nucl Med Mol Imaging (2009) 2.15
A mouse macrophage lipidome. J Biol Chem (2010) 2.14
Exosome-mediated transfer of miR-133b from multipotent mesenchymal stromal cells to neural cells contributes to neurite outgrowth. Stem Cells (2012) 2.14
25-Hydroxycholesterol secreted by macrophages in response to Toll-like receptor activation suppresses immunoglobulin A production. Proc Natl Acad Sci U S A (2009) 2.14
Background mutations in parental cells account for most of the genetic heterogeneity of induced pluripotent stem cells. Cell Stem Cell (2012) 2.11
Knockout of the cholesterol 24-hydroxylase gene in mice reveals a brain-specific mechanism of cholesterol turnover. J Biol Chem (2003) 2.11
Human bone marrow stromal cell treatment improves neurological functional recovery in EAE mice. Exp Neurol (2005) 2.11
Therapeutic benefit of bone marrow stromal cells administered 1 month after stroke. J Cereb Blood Flow Metab (2006) 2.10
Spatial cluster detection for censored outcome data. Biometrics (2007) 2.07
Clinical, virologic, histologic, and biochemical outcomes after successful HCV therapy: a 5-year follow-up of 150 patients. Hepatology (2009) 2.07
p53-mediated senescence impairs the apoptotic response to chemotherapy and clinical outcome in breast cancer. Cancer Cell (2012) 2.04
Purification of nucleic acids by extraction with phenol:chloroform. CSH Protoc (2006) 2.04
mTOR promotes survival and astrocytic characteristics induced by Pten/AKT signaling in glioblastoma. Neoplasia (2005) 2.04
Gene targeting in vivo by adeno-associated virus vectors. Nat Biotechnol (2006) 2.03
A pivotal role for endogenous TGF-beta-activated kinase-1 in the LKB1/AMP-activated protein kinase energy-sensor pathway. Proc Natl Acad Sci U S A (2006) 2.03
Mammalian wax biosynthesis. I. Identification of two fatty acyl-Coenzyme A reductases with different substrate specificities and tissue distributions. J Biol Chem (2004) 2.02
The impact of gene polymorphism and high on-treatment platelet reactivity on clinical follow-up: outcomes in patients with acute coronary syndrome after drug-eluting stent implantation. EuroIntervention (2013) 2.02
Salivary gland tissue expression of interleukin-23 and interleukin-17 in Sjögren's syndrome: findings in humans and mice. Arthritis Rheum (2008) 2.01
Genetic bio-ancestry and social construction of racial classification in social surveys in the contemporary United States. Demography (2014) 2.00
A Papaver somniferum 10-gene cluster for synthesis of the anticancer alkaloid noscapine. Science (2012) 2.00
TLR7-dependent and FcgammaR-independent production of type I interferon in experimental mouse lupus. J Exp Med (2008) 1.99
Engineering of human pluripotent stem cells by AAV-mediated gene targeting. Mol Ther (2010) 1.99
Relationship between the pattern of hepatic iron deposition and histological severity in nonalcoholic fatty liver disease. Hepatology (2010) 1.98
Evidence that an early pregnancy causes a persistent decrease in the number of functional mammary epithelial stem cells--implications for pregnancy-induced protection against breast cancer. Stem Cells (2008) 1.96
Identification of two new loci at IL23R and RAB32 that influence susceptibility to leprosy. Nat Genet (2011) 1.95
Parental decision making about the HPV vaccine. Cancer Epidemiol Biomarkers Prev (2010) 1.94
Transcriptome analysis of root transporters reveals participation of multiple gene families in the response to cation stress. Plant J (2003) 1.93
NF-kappa B-inducing kinase establishes self-tolerance in a thymic stroma-dependent manner. J Immunol (2004) 1.92
Microscopic margins and patterns of treatment failure in resected pancreatic adenocarcinoma. Arch Surg (2012) 1.91
Hippocampal hypometabolism predicts cognitive decline from normal aging. Neurobiol Aging (2007) 1.90
Angiopoietin1/Tie2 and VEGF/Flk1 induced by MSC treatment amplifies angiogenesis and vascular stabilization after stroke. J Cereb Blood Flow Metab (2007) 1.89
De-orphanization of cytochrome P450 2R1: a microsomal vitamin D 25-hydroxilase. J Biol Chem (2003) 1.89
A simple, rapid, high-fidelity and cost-effective PCR-based two-step DNA synthesis method for long gene sequences. Nucleic Acids Res (2004) 1.86
Systemic administration of exosomes released from mesenchymal stromal cells promote functional recovery and neurovascular plasticity after stroke in rats. J Cereb Blood Flow Metab (2013) 1.85
Large-scale analysis of adeno-associated virus vector integration sites in normal human cells. J Virol (2005) 1.85
Human bone marrow stromal cell cultures conditioned by traumatic brain tissue extracts: growth factor production. J Neurosci Res (2002) 1.85