Published in J Virol on January 01, 2002
The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy. Proc Natl Acad Sci U S A (2002) 4.30
Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol (2004) 3.65
Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol (2005) 3.28
Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys. Nat Med (2009) 3.25
Identification of a heparin-binding motif on adeno-associated virus type 2 capsids. J Virol (2003) 3.25
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther (2008) 2.93
Identification of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding. J Virol (2003) 2.93
Alpha2,3 and alpha2,6 N-linked sialic acids facilitate efficient binding and transduction by adeno-associated virus types 1 and 6. J Virol (2006) 2.79
Effect of genome size on AAV vector packaging. Mol Ther (2009) 2.77
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results. Hum Gene Ther (2011) 2.75
Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol (2006) 2.63
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest (2008) 2.40
Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial. Hum Gene Ther (2011) 2.29
Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther (2008) 2.19
Structure of adeno-associated virus serotype 8, a gene therapy vector. J Virol (2007) 2.15
Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol (2007) 2.05
Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps. J Virol (2005) 2.05
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol Ther (2011) 2.04
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol (2010) 2.02
Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis. Proc Natl Acad Sci U S A (2006) 1.97
Structurally mapping the diverse phenotype of adeno-associated virus serotype 4. J Virol (2006) 1.97
Preferential labeling of inhibitory and excitatory cortical neurons by endogenous tropism of adeno-associated virus and lentivirus vectors. Neuroscience (2009) 1.92
A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy. J Transl Med (2007) 1.86
Treatment of human disease by adeno-associated viral gene transfer. Hum Genet (2006) 1.84
A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol Ther (2009) 1.80
Novel AAV serotypes for improved ocular gene transfer. J Gene Med (2008) 1.80
AAV hybrid serotypes: improved vectors for gene delivery. Curr Gene Ther (2005) 1.80
A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction. J Virol (2002) 1.71
Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus. J Virol (2004) 1.67
Tropism and toxicity of adeno-associated viral vector serotypes 1, 2, 5, 6, 7, 8, and 9 in rat neurons and glia in vitro. Virology (2007) 1.66
Characterization of the transcription profile of adeno-associated virus type 5 reveals a number of unique features compared to previously characterized adeno-associated viruses. J Virol (2002) 1.64
Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J Virol (2004) 1.64
Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry. J Virol (2006) 1.64
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
Rapid directional shift of mitochondrial DNA heteroplasmy in animal tissues by a mitochondrially targeted restriction endonuclease. Proc Natl Acad Sci U S A (2005) 1.54
Generation and characterization of chimeric recombinant AAV vectors. Mol Ther (2003) 1.54
Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. J Virol (2007) 1.52
State-of-the-art 2003 on PKU gene therapy. Mol Genet Metab (2004) 1.51
Recombinant adeno-associated virus type 2 pseudotypes: comparing safety, specificity, and transduction efficiency in the primate striatum. Laboratory investigation. J Neurosurg (2010) 1.50
Microvesicle-associated AAV vector as a novel gene delivery system. Mol Ther (2012) 1.49
Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations. Gene Ther (2009) 1.48
Structure of adeno-associated virus type 4. J Virol (2005) 1.47
Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue. Genet Vaccines Ther (2004) 1.47
Recombinant adeno-associated virus transduction and integration. Mol Ther (2008) 1.44
Intracellular transport of recombinant adeno-associated virus vectors. Gene Ther (2012) 1.44
Novel adeno-associated virus vector vaccine restricts replication of simian immunodeficiency virus in macaques. J Virol (2005) 1.36
Comparison of AAV serotypes for gene delivery to dorsal root ganglion neurons. Mol Ther (2010) 1.36
Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery. Mol Ther (2009) 1.35
Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. PLoS One (2013) 1.33
Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system. Mol Ther (2005) 1.30
Characterization of tissue tropism determinants of adeno-associated virus type 1. J Virol (2003) 1.29
Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective. Hum Gene Ther (2015) 1.27
AAV's anatomy: roadmap for optimizing vectors for translational success. Curr Gene Ther (2010) 1.27
Marker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production. J Virol (2003) 1.24
The assembly-activating protein promotes capsid assembly of different adeno-associated virus serotypes. J Virol (2011) 1.23
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
The structure of adeno-associated virus serotype 3B (AAV-3B): insights into receptor binding and immune evasion. Virology (2010) 1.21
Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapy. Mol Ther (2010) 1.21
Detection of intact rAAV particles up to 6 years after successful gene transfer in the retina of dogs and primates. Mol Ther (2008) 1.20
Viral vectors for gene delivery to the central nervous system. Neurobiol Dis (2011) 1.20
Viral vectors: from virology to transgene expression. Br J Pharmacol (2009) 1.19
Adeno-associated virus vectors: potential applications for cancer gene therapy. Cancer Gene Ther (2005) 1.19
Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector. J Virol (2002) 1.19
Comparison of transduction efficiency of recombinant AAV serotypes 1, 2, 5, and 8 in the rat nigrostriatal system. J Neurochem (2009) 1.18
Structural insight into the unique properties of adeno-associated virus serotype 9. J Virol (2012) 1.18
Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium. Mol Ther (2009) 1.18
Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Hum Gene Ther (2005) 1.17
Production, purification, crystallization and preliminary X-ray structural studies of adeno-associated virus serotype 5. Acta Crystallogr Sect F Struct Biol Cryst Commun (2005) 1.17
Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice. Am J Physiol Cell Physiol (2008) 1.16
Gene therapy for muscular dystrophy: lessons learned and path forward. Neurosci Lett (2012) 1.16
Comparative analysis of adeno-associated virus capsid stability and dynamics. J Virol (2013) 1.16
Secretion of a TNFR:Fc fusion protein following pulmonary administration of pseudotyped adeno-associated virus vectors. J Virol (2004) 1.15
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
Induction of robust immune responses against human immunodeficiency virus is supported by the inherent tropism of adeno-associated virus type 5 for dendritic cells. J Virol (2006) 1.13
Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery. Vision Res (2007) 1.12
Infectious molecular clones of adeno-associated virus isolated directly from human tissues. J Virol (2008) 1.11
Optogenetic inhibition of dorsal medial prefrontal cortex attenuates stress-induced reinstatement of palatable food seeking in female rats. J Neurosci (2013) 1.09
Short Promoters in Viral Vectors Drive Selective Expression in Mammalian Inhibitory Neurons, but do not Restrict Activity to Specific Inhibitory Cell-Types. Front Neural Circuits (2009) 1.08
Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum Gene Ther (2010) 1.08
Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse. Exp Eye Res (2010) 1.07
Scalable generation of high-titer recombinant adeno-associated virus type 5 in insect cells. J Virol (2006) 1.07
Rapid, long-term labeling of cells in the developing and adult rodent visual cortex using double-stranded adeno-associated viral vectors. Dev Neurobiol (2009) 1.07
Gene therapy for type I glycogen storage diseases. Curr Gene Ther (2007) 1.07
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. Mol Ther (2013) 1.06
Structure-function analysis of receptor-binding in adeno-associated virus serotype 6 (AAV-6). Virology (2011) 1.05
Different tropism of adenoviruses and adeno-associated viruses to corneal cells: implications for corneal gene therapy. Mol Vis (2008) 1.05
Cardiac gene therapy with SERCA2a: from bench to bedside. J Mol Cell Cardiol (2010) 1.05
Biology of adeno-associated viral vectors in the central nervous system. Front Mol Neurosci (2014) 1.04
Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles. J Virol (2012) 1.04
Surface loop dynamics in adeno-associated virus capsid assembly. J Virol (2008) 1.04
Corticospinal tract transduction: a comparison of seven adeno-associated viral vector serotypes and a non-integrating lentiviral vector. Gene Ther (2011) 1.03
Antiangiogenic gene therapy of cancer: recent developments. J Transl Med (2004) 1.03
Proteolytic mapping of the adeno-associated virus capsid. Mol Ther (2006) 1.03
Production and discovery of novel recombinant adeno-associated viral vectors. Curr Protoc Microbiol (2012) 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
Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria. Gene Ther (2006) 1.02
Structure and dynamics of adeno-associated virus serotype 1 VP1-unique N-terminal domain and its role in capsid trafficking. J Virol (2013) 1.01
Enhanced gene transfer to arthritic joints using adeno-associated virus type 5: implications for intra-articular gene therapy. Ann Rheum Dis (2005) 1.01
Selective extraction of polyoma DNA from infected mouse cell cultures. J Mol Biol (1967) 106.91
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol (1998) 8.45
Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther (1999) 7.96
Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol (1998) 7.76
Nucleotide sequence and organization of the adeno-associated virus 2 genome. J Virol (1983) 7.52
Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol (1989) 6.69
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet (2000) 5.80
Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J Virol (1998) 4.71
Gene therapy vectors based on adeno-associated virus type 1. J Virol (1999) 4.63
Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc Natl Acad Sci U S A (2000) 4.39
Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors. J Virol (2000) 3.84
Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther (1998) 3.77
Cloning and characterization of adeno-associated virus type 5. J Virol (1999) 3.36
Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol Ther (2000) 3.32
Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism. J Virol (2000) 3.27
Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity. J Virol (2001) 3.11
Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J Biol Chem (1993) 3.02
Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J Virol (2000) 3.00
Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles. J Virol (1997) 2.91
Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. Hum Gene Ther (2001) 2.69
Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer. J Virol (2001) 2.46
DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids. EMBO J (2001) 2.43
Subcellular compartmentalization of adeno-associated virus type 2 assembly. J Virol (1997) 2.31
Nucleotide sequencing and generation of an infectious clone of adeno-associated virus 3. Virology (1996) 2.26
Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J Virol (2000) 2.26
Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nat Med (1999) 2.24
Role for highly regulated rep gene expression in adeno-associated virus vector production. J Virol (1997) 2.19
Adeno-associated virus type 2-mediated gene transfer: altered endocytic processing enhances transduction efficiency in murine fibroblasts. J Virol (2001) 2.03
Human adeno-associated virus type 5 is only distantly related to other known primate helper-dependent parvoviruses. J Virol (1999) 1.94
Factors influencing recombinant adeno-associated virus production. Hum Gene Ther (1998) 1.86
Colocalization of adeno-associated virus Rep and capsid proteins in the nuclei of infected cells. J Virol (1992) 1.86
Insertional mutagenesis of AAV2 capsid and the production of recombinant virus. Virology (1999) 1.76
Adeno-associated virus (AAV)-3-based vectors transduce haematopoietic cells not susceptible to transduction with AAV-2-based vectors. J Gen Virol (2000) 1.73
Adeno-associated virus expression systems for gene transfer. Curr Opin Biotechnol (1998) 1.66
The Rep52 gene product of adeno-associated virus is a DNA helicase with 3'-to-5' polarity. J Virol (1998) 1.61
Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'gamma)2 antibody. Nat Biotechnol (1999) 1.51
AAV vectors: is clinical success on the horizon? Gene Ther (2000) 1.47
Adeno-associated virus vectors for gene therapy: more pros than cons? Mol Med Today (2000) 1.45
Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element. J Virol (2000) 1.32
Control of adeno-associated virus type 2 cap gene expression: relative influence of helper virus, terminal repeats, and Rep proteins. J Virol (1997) 1.28
Incorporation of adeno-associated virus in a calcium phosphate coprecipitate improves gene transfer to airway epithelia in vitro and in vivo. J Virol (2000) 1.20
Inhibition of PrKX, a novel protein kinase, and the cyclic AMP-dependent protein kinase PKA by the regulatory proteins of adeno-associated virus type 2. Mol Cell Biol (1998) 1.20
Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood (2000) 1.19
Amino-terminal domain exchange redirects origin-specific interactions of adeno-associated virus rep78 in vitro. J Virol (2001) 1.09
Evaluation of adeno-associated virus-mediated gene transfer into the rat retina by clinical fluorescence photography. Hum Gene Ther (1999) 1.02
A modified double antibody sandwich enzyme-linked immunosorbent assay for measurement of alpha-1-antitrypsin in biologic fluids. J Immunol Methods (1985) 0.89
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol (2005) 5.27
Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther (2006) 4.36
Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med (2010) 4.28
Production and characterization of adeno-associated viral vectors. Nat Protoc (2006) 4.11
Investigation of the cause of death in a gene-therapy trial. N Engl J Med (2009) 3.02
Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther (2011) 2.91
Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat Med (2003) 2.70
Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol (2006) 2.63
microRNA-1 and microRNA-206 regulate skeletal muscle satellite cell proliferation and differentiation by repressing Pax7. J Cell Biol (2010) 2.57
The AAV vector toolkit: poised at the clinical crossroads. Mol Ther (2012) 2.53
Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet (2004) 2.36
Critical role of calpain I in mitochondrial release of apoptosis-inducing factor in ischemic neuronal injury. J Neurosci (2007) 2.23
Historical variations in mutation rate in an epidemic pathogen, Yersinia pestis. Proc Natl Acad Sci U S A (2012) 2.22
Remodeling of cortical bone allografts mediated by adherent rAAV-RANKL and VEGF gene therapy. Nat Med (2005) 2.10
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol (2009) 2.07
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer. Proc Natl Acad Sci U S A (2008) 2.06
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Arachidonic acid epoxygenase metabolites stimulate endothelial cell growth and angiogenesis via mitogen-activated protein kinase and phosphatidylinositol 3-kinase/Akt signaling pathways. J Pharmacol Exp Ther (2005) 2.05
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol Ther (2011) 2.04
Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Hum Gene Ther (2011) 1.90
Cytochrome P450 2J2 promotes the neoplastic phenotype of carcinoma cells and is up-regulated in human tumors. Cancer Res (2005) 1.86
A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection. Proc Natl Acad Sci U S A (2009) 1.85
Second-generation tetracycline-regulatable promoter: repositioned tet operator elements optimize transactivator synergy while shorter minimal promoter offers tight basal leakiness. J Gene Med (2004) 1.80
AAV hybrid serotypes: improved vectors for gene delivery. Curr Gene Ther (2005) 1.80
Increased atherosclerotic lesions in apoE mice with plasma phospholipid transfer protein overexpression. Arterioscler Thromb Vasc Biol (2003) 1.79
Inflammatory markers and risk of type 2 diabetes: a systematic review and meta-analysis. Diabetes Care (2013) 1.77
Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX. Blood (2008) 1.72
Characterization of genome integrity for oversized recombinant AAV vector. Mol Ther (2009) 1.71
Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice. Hum Gene Ther (2008) 1.65
Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J Virol (2004) 1.64
Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle. Mol Ther (2008) 1.63
Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. Blood (2003) 1.62
Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol Ther (2009) 1.61
Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice. Hum Gene Ther (2002) 1.56
Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs. Mol Ther (2010) 1.56
Cytochrome p450 epoxygenase promotes human cancer metastasis. Cancer Res (2007) 1.55
Generation and characterization of chimeric recombinant AAV vectors. Mol Ther (2003) 1.54
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther (2013) 1.54
Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery. Hum Gene Ther (2011) 1.53
Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc Natl Acad Sci U S A (2009) 1.53
Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. J Virol (2007) 1.52
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Enhancement of adeno-associated virus infection by mobilizing capsids into and out of the nucleolus. J Virol (2008) 1.50
Aryl Hydrocarbon Receptor Activation Down-Regulates IL-7 and Reduces Inflammation in a Mouse Model of DSS-Induced Colitis. Dig Dis Sci (2015) 1.50
Curcumin prevents chronic alcohol-induced liver disease involving decreasing ROS generation and enhancing antioxidative capacity. Phytomedicine (2012) 1.48
Widespread and stable pancreatic gene transfer by adeno-associated virus vectors via different routes. Diabetes (2006) 1.45
Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle. J Virol (2008) 1.44
Host cell DNA repair pathways in adeno-associated viral genome processing. J Virol (2006) 1.43
Sustained whole-body functional rescue in congestive heart failure and muscular dystrophy hamsters by systemic gene transfer. Circulation (2005) 1.42
Structural characterization of the dual glycan binding adeno-associated virus serotype 6. J Virol (2010) 1.42
Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain. Mol Ther (2008) 1.41
Lamin A/C-mediated neuromuscular junction defects in Emery-Dreifuss muscular dystrophy. J Cell Biol (2009) 1.36
Characterization of tissue tropism determinants of adeno-associated virus type 1. J Virol (2003) 1.29
Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies. Mamm Genome (2012) 1.28
Comparative transduction efficiency of AAV vector serotypes 1-6 in the substantia nigra and striatum of the primate brain. Mol Ther (2009) 1.28
Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle. Hum Gene Ther (2010) 1.26
One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice. Mol Ther (2010) 1.25
DOT1L regulates dystrophin expression and is critical for cardiac function. Genes Dev (2011) 1.25
Long-term doxycycline-regulated transgene expression in the retina of nonhuman primates following subretinal injection of recombinant AAV vectors. Mol Ther (2006) 1.24
Marker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production. J Virol (2003) 1.24
Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application. Mol Ther (2010) 1.24
In vivo gene regulation using tetracycline-regulatable systems. Adv Drug Deliv Rev (2009) 1.23
Long-term follow-up after gene therapy for canavan disease. Sci Transl Med (2012) 1.22
Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle. Mol Ther (2009) 1.21
Mutagenesis of adeno-associated virus type 2 capsid protein VP1 uncovers new roles for basic amino acids in trafficking and cell-specific transduction. J Virol (2010) 1.21
Hydrodynamic limb vein injection of adeno-associated virus serotype 8 vector carrying canine myostatin propeptide gene into normal dogs enhances muscle growth. Hum Gene Ther (2009) 1.21
Adeno-associated virus (AAV) gene therapy for neurological disease. Neuropharmacology (2012) 1.21
Detection of intact rAAV particles up to 6 years after successful gene transfer in the retina of dogs and primates. Mol Ther (2008) 1.20
Viral vectors for gene delivery to the central nervous system. Neurobiol Dis (2011) 1.20
Amelioration of laminin-alpha2-deficient congenital muscular dystrophy by somatic gene transfer of miniagrin. Proc Natl Acad Sci U S A (2005) 1.19
Adeno-associated virus vectorology, manufacturing, and clinical applications. Methods Enzymol (2012) 1.19
Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector. J Virol (2002) 1.19
Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium. Mol Ther (2009) 1.18
Systemic human minidystrophin gene transfer improves functions and life span of dystrophin and dystrophin/utrophin-deficient mice. J Orthop Res (2009) 1.17
Intracellular viral processing, not single-stranded DNA accumulation, is crucial for recombinant adeno-associated virus transduction. J Virol (2004) 1.17
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther (2013) 1.16
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. Mol Ther (2012) 1.16
Little vector, big gene transduction: fragmented genome reassembly of adeno-associated virus. Mol Ther (2010) 1.16
Expression of human factor VIII by splicing between dimerized AAV vectors. Mol Ther (2002) 1.16
KLF8 involves in TGF-beta-induced EMT and promotes invasion and migration in gastric cancer cells. J Cancer Res Clin Oncol (2013) 1.16
Effects of adeno-associated virus DNA hairpin structure on recombination. J Virol (2005) 1.15
A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and -5. Mol Ther (2002) 1.14
Expression of intercellular adhesion molecule 1 by hepatocellular carcinoma stem cells and circulating tumor cells. Gastroenterology (2013) 1.14
Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins. Proc Natl Acad Sci U S A (2011) 1.14
Secretion of extracellular superoxide dismutase from muscle transduced with recombinant adenovirus inhibits the growth of B16 melanomas in mice. Mol Cancer Res (2003) 1.14
Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette. Hum Gene Ther (2008) 1.13
Loss of enteral nutrition in a mouse model results in intestinal epithelial barrier dysfunction. Ann N Y Acad Sci (2012) 1.13
Enhancing muscle membrane repair by gene delivery of MG53 ameliorates muscular dystrophy and heart failure in δ-Sarcoglycan-deficient hamsters. Mol Ther (2012) 1.13
Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction. Proc Natl Acad Sci U S A (2007) 1.12
Characterizing species abundance distributions across taxa and ecosystems using a simple maximum entropy model. Ecology (2012) 1.12
Potential of DNMT and its Epigenetic Regulation for Lung Cancer Therapy. Curr Genomics (2009) 1.11
miR-375 regulates rat alveolar epithelial cell trans-differentiation by inhibiting Wnt/β-catenin pathway. Nucleic Acids Res (2013) 1.11
Self-complementary AAV2.5-BMP2-coated femoral allografts mediated superior bone healing versus live autografts in mice with equivalent biomechanics to unfractured femur. Mol Ther (2011) 1.10