Published in PLoS One on March 13, 2012
Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8. Hum Gene Ther (2013) 0.82
Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial. Hum Gene Ther (2015) 0.81
A gene-expression screen identifies a non-toxic sumoylation inhibitor that mimics SUMO-less human LRH-1 in liver. Elife (2015) 0.81
Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease. Mol Ther (2016) 0.75
Overexpression and deletion of phospholipid transfer protein reduce HDL mass and cholesterol efflux capacity but not macrophage reverse cholesterol transport. J Lipid Res (2017) 0.75
Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI. Mol Ther Methods Clin Dev (2017) 0.75
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol (2007) 5.36
Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol (1999) 5.35
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet (2011) 4.67
Distribution of organelles and membranes between hepatocytes and nonhepatocytes in the rat liver parenchyma. A stereological study. J Cell Biol (1977) 4.13
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest (2003) 3.52
Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol (2001) 3.38
Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med (2006) 3.22
Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J Virol (1998) 3.14
Mannose 6-phosphate receptors and lysosomal enzyme targeting. J Biol Chem (1989) 2.77
Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J Virol (1998) 2.40
Antigen-presenting cell function in the tolerogenic liver environment. Nat Rev Immunol (2010) 2.38
Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet (2004) 2.36
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood (2007) 2.28
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther (2009) 2.08
Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol Ther (2000) 2.01
Adeno-associated virus as a vector for liver-directed gene therapy. J Virol (1998) 1.94
Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc Natl Acad Sci U S A (1999) 1.90
Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II. Mol Ther (2005) 1.87
Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol Ther (2009) 1.85
High levels of transduction of human dendritic cells with optimized SIV vectors. Mol Ther (2002) 1.73
Gene delivery to the juvenile mouse liver using AAV2/8 vectors. Mol Ther (2008) 1.73
Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol Ther (2000) 1.71
Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy. Hum Gene Ther (1999) 1.70
Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. Mol Ther (2006) 1.64
Lentivirally transduced dendritic cells as a tool for cancer immunotherapy. J Gene Med (2003) 1.59
Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain. Gene Ther (2000) 1.59
Autophagy, mitochondria and cell death in lysosomal storage diseases. Autophagy (2007) 1.55
Hepatic gene transfer as a means of tolerance induction to transgene products. Curr Gene Ther (2009) 1.54
Lysosomal storage diseases as disorders of autophagy. Autophagy (2007) 1.46
Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression. Gene Ther (2006) 1.39
Cell proliferation and renewal of normal hepatocytes and bile duct cells in adult mouse liver. Liver (2002) 1.34
Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element. J Virol (2000) 1.32
Immunology of the healthy liver: old questions and new insights. Gastroenterology (2001) 1.25
Self-inactivating retroviral vectors with improved RNA processing. Gene Ther (2004) 1.23
Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle. Mol Ther (2007) 1.21
Hemophilia gene therapy: a Holy Grail found. Mol Ther (2011) 1.20
Adenoviral-mediated, high-level, cell-specific transgene expression: a SYN1-WPRE cassette mediates increased transgene expression with no loss of neuron specificity. Mol Ther (2002) 1.13
Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus. Mol Ther (2004) 1.12
Comparison of gene expression after intraperitoneal delivery of AAV2 or AAV5 in utero. Mol Ther (2003) 1.12
Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig. Mol Ther (2006) 1.11
Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors. Proc Natl Acad Sci U S A (2009) 1.11
Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Ther (2005) 1.09
Optimization of the human factor VIII complementary DNA expression plasmid for gene therapy of hemophilia A. Blood Coagul Fibrinolysis (1997) 1.08
AAV vectors avoid inflammatory signals necessary to render transduced hepatocyte targets for destructive T cells. Mol Ther (2010) 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
Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction. Hum Gene Ther (2010) 1.07
Abnormal autophagy, ubiquitination, inflammation and apoptosis are dependent upon lysosomal storage and are useful biomarkers of mucopolysaccharidosis VI. Pathogenetics (2009) 1.06
Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer. Mol Ther (2010) 1.06
Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice. Mol Ther (2007) 1.06
WPRE-mediated enhancement of gene expression is promoter and cell line specific. Gene (2006) 0.98
Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses. Hum Gene Ther (2010) 0.98
Cellular immunotherapy and autologous transplantation for hematologic malignancy. Immunol Rev (1997) 0.96
Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice. Thromb Res (2006) 0.95
Transient expression of genes delivered to newborn rat liver using recombinant adeno-associated virus 2/8 vectors. J Gene Med (2009) 0.93
Measurements of vector-derived neurotrophic factor and green fluorescent protein levels in the brain. Methods (2002) 0.92
The hepatic immune system. Crit Rev Immunol (2002) 0.90
Controlled delivery of glial cell line-derived neurotrophic factor by a single tetracycline-inducible AAV vector. Exp Neurol (2007) 0.90
Genetic analysis of the antibody response to AAV2 and factor IX. Mol Ther (2005) 0.90
Mitochondrial nitric oxide synthase drives redox signals for proliferation and quiescence in rat liver development. Hepatology (2004) 0.89
Woodchuck hepatitis virus post-transcriptional regulation element enhances transgene expression from adenovirus vectors. Biochim Biophys Acta (2003) 0.86
B-cell-specific transgene expression using a self-inactivating retroviral vector with human CD19 promoter and viral post-transcriptional regulatory element. Gene Ther (2004) 0.86
Optimization of transgene expression at the posttranscriptional level in neural cells: implications for gene therapy. Mol Ther (2003) 0.86
Persistent liver expression of murine apoA-l using vectors based on adeno-associated viral vectors serotypes 5 and 1. Atherosclerosis (2005) 0.85
Long-term transgene expression can be mediated in the brain by adenoviral vectors when powerful neuron-specific promoters are used. J Gene Med (2003) 0.85
Perinatal gene transfer to the liver. Curr Pharm Des (2011) 0.84
Posttranscriptional regulatory elements enhance antigen expression and DNA vaccine efficacy. DNA Cell Biol (2009) 0.83
Targeting adenoviral transgene expression to neurons. Mol Cell Neurosci (2008) 0.83
A novel combination of promoter and enhancers increases transgene expression in vascular smooth muscle cells in vitro and coronary arteries in vivo after adenovirus-mediated gene transfer. Gene Ther (2003) 0.82
Effect of posttranscriptional regulatory elements on transgene expression and virus production in the context of retrovirus vectors. Virology (2005) 0.82
Enhanced inhibition of human immunodeficiency virus type 1 replication by novel lentiviral vectors expressing human immunodeficiency virus type 1 envelope antisense RNA. Hum Gene Ther (2002) 0.81
Hepadna virus integration generates virus-cell cotranscripts carrying 3' truncated X genes in human and woodchuck liver tumors. J Med Virol (1995) 0.81
Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med (2008) 17.21
A gene network regulating lysosomal biogenesis and function. Science (2009) 7.17
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet (2009) 6.71
A high-resolution anatomical atlas of the transcriptome in the mouse embryo. PLoS Biol (2011) 4.84
Human chromosome 21 gene expression atlas in the mouse. Nature (2002) 4.36
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther (2009) 3.93
MicroRNAs involved in molecular circuitries relevant for the Duchenne muscular dystrophy pathogenesis are controlled by the dystrophin/nNOS pathway. Cell Metab (2010) 2.99
AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med (2012) 2.87
Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients. Invest Ophthalmol Vis Sci (2007) 2.69
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest (2008) 2.40
Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport. J Clin Invest (2004) 2.30
MicroRNA target prediction by expression analysis of host genes. Genome Res (2008) 2.21
UTRdb and UTRsite (RELEASE 2010): a collection of sequences and regulatory motifs of the untranslated regions of eukaryotic mRNAs. Nucleic Acids Res (2009) 2.11
Identification and characterization of microRNAs expressed in the mouse eye. Invest Ophthalmol Vis Sci (2007) 2.05
Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol (2007) 2.05
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology (2013) 1.85
Development of a genotyping microarray for Usher syndrome. J Med Genet (2006) 1.63
miRNeye: a microRNA expression atlas of the mouse eye. BMC Genomics (2010) 1.59
Slc7a7 disruption causes fetal growth retardation by downregulating Igf1 in the mouse model of lysinuric protein intolerance. Am J Physiol Cell Physiol (2007) 1.59
Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. Proc Natl Acad Sci U S A (2006) 1.55
Recessive mutations in SLC38A8 cause foveal hypoplasia and optic nerve misrouting without albinism. Am J Hum Genet (2013) 1.53
The human visual cortex responds to gene therapy-mediated recovery of retinal function. J Clin Invest (2011) 1.52
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Identification of microRNA-regulated gene networks by expression analysis of target genes. Genome Res (2012) 1.51
miR-204 is required for lens and retinal development via Meis2 targeting. Proc Natl Acad Sci U S A (2010) 1.48
RNA interference-mediated suppression and replacement of human rhodopsin in vivo. Am J Hum Genet (2007) 1.47
Versatility of AAV vectors for retinal gene transfer. Vision Res (2007) 1.44
Natural antisense transcripts associated with genes involved in eye development. Hum Mol Genet (2005) 1.40
Vax2 inactivation in mouse determines alteration of the eye dorsal-ventral axis, misrouting of the optic fibres and eye coloboma. Development (2002) 1.39
Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration. Mol Ther (2004) 1.35
microRNAs and genetic diseases. Pathogenetics (2009) 1.32
A homozygous missense mutation in the IRBP gene (RBP3) associated with autosomal recessive retinitis pigmentosa. Invest Ophthalmol Vis Sci (2008) 1.27
Bile acids modulate tight junction structure and barrier function of Caco-2 monolayers via EGFR activation. Am J Physiol Gastrointest Liver Physiol (2008) 1.25
A 76-kb duplicon maps close to the BCR gene on chromosome 22 and the ABL gene on chromosome 9: possible involvement in the genesis of the Philadelphia chromosome translocation. Proc Natl Acad Sci U S A (2002) 1.25
Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery. Hum Mol Genet (2006) 1.21
Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle. Mol Ther (2007) 1.21
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
Mutations in IMPG2, encoding interphotoreceptor matrix proteoglycan 2, cause autosomal-recessive retinitis pigmentosa. Am J Hum Genet (2010) 1.19
Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice. Hum Gene Ther (2008) 1.17
The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts. Mol Ther (2009) 1.17
AAV-mediated gene transfer for retinal diseases. Expert Opin Biol Ther (2006) 1.15
PEGylation of a vesicular stomatitis virus G pseudotyped lentivirus vector prevents inactivation in serum. J Virol (2004) 1.15
Immunoglobulin A anti-tissue transglutaminase antibody deposits in the small intestinal mucosa of children with no villous atrophy. J Pediatr Gastroenterol Nutr (2008) 1.13
An autoregulatory loop directs the tissue-specific expression of p63 through a long-range evolutionarily conserved enhancer. Mol Cell Biol (2006) 1.13
Filamin A is mutated in X-linked chronic idiopathic intestinal pseudo-obstruction with central nervous system involvement. Am J Hum Genet (2007) 1.13
A novel peroxisome proliferator-activated receptor gamma isoform with dominant negative activity generated by alternative splicing. J Biol Chem (2005) 1.12
Pax6 regulates gene expression in the vertebrate lens through miR-204. PLoS Genet (2013) 1.11
Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice. Hum Gene Ther (2006) 1.11
The C. elegans HP1 homologue HPL-2 and the LIN-13 zinc finger protein form a complex implicated in vulval development. Dev Biol (2006) 1.10
Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments. PLoS One (2009) 1.09
Correlation between photoreceptor layer integrity and visual function in patients with Stargardt disease: implications for gene therapy. Invest Ophthalmol Vis Sci (2012) 1.09
The long noncoding RNA Vax2os1 controls the cell cycle progression of photoreceptor progenitors in the mouse retina. RNA (2011) 1.08
Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates. Hum Gene Ther (2005) 1.07
Androgen-induced cell migration: role of androgen receptor/filamin A association. PLoS One (2011) 1.06
Abnormal autophagy, ubiquitination, inflammation and apoptosis are dependent upon lysosomal storage and are useful biomarkers of mucopolysaccharidosis VI. Pathogenetics (2009) 1.06
Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer. Mol Ther (2010) 1.06
BBS1 mutations in a wide spectrum of phenotypes ranging from nonsyndromic retinitis pigmentosa to Bardet-Biedl syndrome. Arch Ophthalmol (2012) 1.06
Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy. PLoS One (2011) 1.05
Hormone-dependent nuclear export of estradiol receptor and DNA synthesis in breast cancer cells. J Cell Biol (2008) 1.05
Effective delivery of large genes to the retina by dual AAV vectors. EMBO Mol Med (2013) 1.04
Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor. Diabetes (2005) 1.04
SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies. Biochem J (2007) 1.02
Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transduction. J Virol (2003) 1.02
Role of atypical protein kinase C in estradiol-triggered G1/S progression of MCF-7 cells. Mol Cell Biol (2004) 1.02
Androgen-stimulated DNA synthesis and cytoskeletal changes in fibroblasts by a nontranscriptional receptor action. J Cell Biol (2003) 1.02
Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa. EMBO Mol Med (2011) 1.02
Molecular diagnosis of Usher syndrome: application of two different next generation sequencing-based procedures. PLoS One (2012) 1.01
Adeno-associated viral vectors for retinal gene transfer. Prog Retin Eye Res (2003) 0.99
Inducible gene expression systems and plant biotechnology. Biotechnol Adv (2009) 0.99
Evaluation of Italian patients with leber congenital amaurosis due to AIPL1 mutations highlights the potential applicability of gene therapy. Invest Ophthalmol Vis Sci (2011) 0.99
Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder. Mol Ther (2011) 0.98
Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses. Hum Gene Ther (2010) 0.98
AAV-mediated gene replacement, either alone or in combination with physical and pharmacological agents, results in partial and transient protection from photoreceptor degeneration associated with betaPDE deficiency. Invest Ophthalmol Vis Sci (2011) 0.97
Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model. Mol Ther (2006) 0.97
CoGemiR: a comparative genomics microRNA database. BMC Genomics (2008) 0.97
Molecular epidemiology of Usher syndrome in Italy. Mol Vis (2011) 0.97
HOCTAR database: a unique resource for microRNA target prediction. Gene (2011) 0.96
Amelioration of both functional and morphological abnormalities in the retina of a mouse model of ocular albinism following AAV-mediated gene transfer. Mol Ther (2005) 0.96
Mutations in IMPG1 cause vitelliform macular dystrophies. Am J Hum Genet (2013) 0.96
Promiscuity of enhancer, coding and non-coding transcription functions in ultraconserved elements. BMC Genomics (2010) 0.95
A high-resolution RNA expression atlas of retinitis pigmentosa genes in human and mouse retinas. Invest Ophthalmol Vis Sci (2008) 0.95
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy. EMBO Mol Med (2012) 0.95
Identification and expression analysis of novel Jakmip1 transcripts. Gene (2007) 0.95
Ocular gene therapy: current progress and future prospects. Trends Mol Med (2008) 0.94
Identification and characterization of C1orf36, a transcript highly expressed in photoreceptor cells, and mutation analysis in retinitis pigmentosa. Biochem Biophys Res Commun (2003) 0.94
Highly conserved elements discovered in vertebrates are present in non-syntenic loci of tunicates, act as enhancers and can be transcribed during development. Nucleic Acids Res (2013) 0.94