Published in Mol Ther on October 01, 2004
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol (2008) 6.82
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther (2006) 4.64
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
A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol Ther (2008) 2.22
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Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery. Mol Ther (2009) 1.35
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. Mol Ther (2010) 1.35
Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol Ther (2008) 1.34
Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. Mol Ther (2008) 1.29
Capsid-expressing DNA in AAV vectors and its elimination by use of an oversize capsid gene for vector production. Gene Ther (2010) 1.29
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Epidermal growth factor receptor is a co-receptor for adeno-associated virus serotype 6. Nat Med (2010) 1.22
Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol Ther (2010) 1.21
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Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature. Mol Ther (2009) 1.13
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Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging. Mol Ther (2009) 1.11
Follistatin-mediated skeletal muscle hypertrophy is regulated by Smad3 and mTOR independently of myostatin. J Cell Biol (2012) 1.10
Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments. PLoS One (2009) 1.09
Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy. Mol Ther (2008) 1.09
Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs. Mol Ther (2010) 1.07
Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease. Gene Ther (2010) 1.06
Skeletal muscle contractions induce acute changes in cytosolic superoxide, but slower responses in mitochondrial superoxide and cellular hydrogen peroxide. PLoS One (2014) 1.06
High-efficiency promoter-dependent transduction by adeno-associated virus type 6 vectors in mouse lung. Hum Gene Ther (2007) 1.05
Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease. Gene Ther (2011) 1.04
Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles. J Virol (2012) 1.04
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Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease. Mol Ther (2013) 0.99
miR-206 represses hypertrophy of myogenic cells but not muscle fibers via inhibition of HDAC4. PLoS One (2013) 0.99
Validation of ultrasonography for non-invasive assessment of diaphragm function in muscular dystrophy. J Physiol (2016) 0.97
AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy. Mol Ther (2011) 0.97
A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery. Gene Ther (2009) 0.97
Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells. Mol Ther Methods Clin Dev (2014) 0.92
Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression. Mol Ther (2009) 0.90
Double oxygen-sensing vector system for robust hypoxia/ischemia-regulated gene induction in cardiac muscle in vitro and in vivo. Mol Ther (2008) 0.89
In Vivo Hepatic Reprogramming of Myofibroblasts with AAV Vectors as a Therapeutic Strategy for Liver Fibrosis. Cell Stem Cell (2016) 0.88
Immune Responses to rAAV6: The Influence of Canine Parvovirus Vaccination and Neonatal Administration of Viral Vector. Front Microbiol (2011) 0.88
Heparin-binding correlates with increased efficiency of AAV1- and AAV6-mediated transduction of striated muscle, but negatively impacts CNS transduction. Gene Ther (2012) 0.88
New vectors and strategies for cardiovascular gene therapy. Curr Gene Ther (2007) 0.87
Gene therapy: light is finally in the tunnel. Protein Cell (2012) 0.87
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy. Nat Commun (2017) 0.86
Genome-wide identification of FoxO-dependent gene networks in skeletal muscle during C26 cancer cachexia. BMC Cancer (2014) 0.86
The potential of sarcospan in adhesion complex replacement therapeutics for the treatment of muscular dystrophy. FEBS J (2013) 0.85
Development of novel activin-targeted therapeutics. Mol Ther (2014) 0.85
Age-dependent dystrophin loss and genetic reconstitution establish a molecular link between dystrophin and heart performance during aging. Mol Ther (2011) 0.85
AAV-6 mediated efficient transduction of mouse lower airways. Virology (2011) 0.84
Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders. Gene Ther (2009) 0.84
Sites in the AAV5 capsid tolerant to deletions and tandem duplications. Arch Biochem Biophys (2010) 0.83
Genetic manipulation of myoblasts and a novel primary myosatellite cell culture system: comparing and optimizing approaches. FEBS J (2013) 0.82
Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy. Hum Mol Genet (2015) 0.80
A needleless liquid jet injection delivery method for cardiac gene therapy: a comparative evaluation versus standard routes of delivery reveals enhanced therapeutic retention and cardiac specific gene expression. J Cardiovasc Transl Res (2014) 0.80
Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9. Skelet Muscle (2015) 0.80
Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering. J Biomed Sci (2014) 0.80
Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site. J Virol (2016) 0.79
RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice. Mol Ther Nucleic Acids (2014) 0.78
Rescue from respiratory dysfunction by transduction of full-length dystrophin to diaphragm via the peritoneal cavity in utrophin/dystrophin double knockout mice. Mol Ther (2011) 0.78
Targeted In Situ Gene Correction of Dysfunctional APOE Alleles to Produce Atheroprotective Plasma ApoE3 Protein. Cardiol Res Pract (2012) 0.78
Transduction of skeletal muscles with common reporter genes can promote muscle fiber degeneration and inflammation. PLoS One (2012) 0.77
Cardiac AAV9 Gene Delivery Strategies in Adult Canines: Assessment by Long-term Serial SPECT Imaging of Sodium Iodide Symporter Expression. Mol Ther (2015) 0.77
Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer. Mol Ther (2015) 0.77
Using AAV vectors expressing the β2-adrenoceptor or associated Gα proteins to modulate skeletal muscle mass and muscle fibre size. Sci Rep (2016) 0.77
Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences. PLoS One (2015) 0.77
AAV6-mediated Cardiac-specific Overexpression of Ribonucleotide Reductase Enhances Myocardial Contractility. Mol Ther (2015) 0.77
Forced expression of muscle specific kinase slows postsynaptic acetylcholine receptor loss in a mouse model of MuSK myasthenia gravis. Physiol Rep (2015) 0.76
Modulating myosin restores muscle function in a mouse model of nemaline myopathy. Ann Neurol (2016) 0.76
Systemic RNAi delivery to the muscles of ROSA26 mice reduces lacZ expression. PLoS One (2014) 0.76
Myocardial Contractile Dysfunction Is Present without Histopathology in a Mouse Model of Limb-Girdle Muscular Dystrophy-2F and Is Prevented after Claudin-5 Virotherapy. Front Physiol (2016) 0.75
Inexpensive, serotype-independent protocol for native and bioengineered recombinant adeno-associated virus purification. J Biol Methods (2016) 0.75
Extracorporeal delivery of rAAV with metabolic exchange and oxygenation. Sci Rep (2013) 0.75
Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies. Clin Lipidol (2010) 0.75
Recombinant Adeno-Associated Virus Serotype 6 (rAAV6) Potently and Preferentially Transduces Rat Astrocytes In vitro and In vivo. Front Cell Neurosci (2016) 0.75
Scalable downstream strategies for purification of recombinant adeno- associated virus vectors in light of the properties. Curr Pharm Biotechnol (2015) 0.75
Satellite Cells in Muscular Dystrophy - Lost in Polarity. Trends Mol Med (2016) 0.75
Distribution of myosin heavy chain isoforms in muscular dystrophy: insights into disease pathology. Musculoskelet Regen (2016) 0.75
Liquid jet delivery method featuring S100A1 gene therapy in the rodent model following acute myocardial infarction. Gene Ther (2015) 0.75
Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med (2004) 5.59
Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells. Nat Cell Biol (2007) 5.50
Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med (2002) 5.07
RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med (2004) 4.63
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A (2008) 4.53
The bifunctional microRNA miR-9/miR-9* regulates REST and CoREST and is downregulated in Huntington's disease. J Neurosci (2008) 3.62
Multiple integrated copies and high-level production of the human retrovirus XMRV (xenotropic murine leukemia virus-related virus) from 22Rv1 prostate carcinoma cells. J Virol (2009) 3.16
rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med (2006) 3.13
Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy. J Clin Invest (2009) 2.89
Sarcolemma-localized nNOS is required to maintain activity after mild exercise. Nature (2008) 2.87
Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression. Mol Ther (2007) 2.76
Foamy virus vector integration sites in normal human cells. Proc Natl Acad Sci U S A (2006) 2.52
Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation (2003) 2.22
Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol (2005) 2.16
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther (2005) 2.08
TGF-beta regulates miR-206 and miR-29 to control myogenic differentiation through regulation of HDAC4. J Biol Chem (2011) 2.06
Suppression of microRNA-29 expression by TGF-β1 promotes collagen expression and renal fibrosis. J Am Soc Nephrol (2011) 2.06
DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo. Ann Neurol (2010) 1.89
Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure. Mol Ther (2007) 1.85
Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors. Hum Gene Ther (2006) 1.78
Therapeutic inhibition of the miR-34 family attenuates pathological cardiac remodeling and improves heart function. Proc Natl Acad Sci U S A (2012) 1.72
Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy. Hum Gene Ther (2007) 1.70
Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat Biotechnol (2002) 1.69
Phosphatidylserine is not the cell surface receptor for vesicular stomatitis virus. J Virol (2004) 1.66
Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol Ther (2007) 1.65
The value of mammalian models for duchenne muscular dystrophy in developing therapeutic strategies. Curr Top Dev Biol (2008) 1.62
Mechanism of cell entry and transformation by enzootic nasal tumor virus. J Virol (2002) 1.61
Sheep retrovirus structural protein induces lung tumours. Nature (2005) 1.61
Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors. Nat Med (2007) 1.58
CHIP suppresses polyglutamine aggregation and toxicity in vitro and in vivo. J Neurosci (2005) 1.57
Dystrophin deficiency in Drosophila reduces lifespan and causes a dilated cardiomyopathy phenotype. Aging Cell (2008) 1.52
Dissecting the signaling and mechanical functions of the dystrophin-glycoprotein complex. J Cell Sci (2006) 1.51
Adeno-associated virus types 5 and 6 use distinct receptors for cell entry. Hum Gene Ther (2006) 1.50
Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. Proc Natl Acad Sci U S A (2002) 1.47
Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle. Mol Ther (2008) 1.47
Activation of dopamine neurons is critical for aversive conditioning and prevention of generalized anxiety. Nat Neurosci (2011) 1.46
Recombinant adeno-associated virus transduction and integration. Mol Ther (2008) 1.44
Envelope-induced cell transformation by ovine betaretroviruses. J Virol (2002) 1.44
Functional deficits in nNOSmu-deficient skeletal muscle: myopathy in nNOS knockout mice. PLoS One (2008) 1.42
Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation. J Clin Invest (2002) 1.41
Role of virus receptor Hyal2 in oncogenic transformation of rodent fibroblasts by sheep betaretrovirus env proteins. J Virol (2003) 1.38
Prevention of muscle aging by myofiber-associated satellite cell transplantation. Sci Transl Med (2010) 1.36
Hyaluronidase 2 negatively regulates RON receptor tyrosine kinase and mediates transformation of epithelial cells by jaagsiekte sheep retrovirus. Proc Natl Acad Sci U S A (2003) 1.35
Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol Ther (2008) 1.34
Connecdenn, a novel DENN domain-containing protein of neuronal clathrin-coated vesicles functioning in synaptic vesicle endocytosis. J Neurosci (2006) 1.33
Single-strand nicks induce homologous recombination with less toxicity than double-strand breaks using an AAV vector template. Nucleic Acids Res (2010) 1.32
Putative phosphatidylinositol 3-kinase (PI3K) binding motifs in ovine betaretrovirus Env proteins are not essential for rodent fibroblast transformation and PI3K/Akt activation. J Virol (2003) 1.30
Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. Mol Ther (2008) 1.29
Emerging strategies for cell and gene therapy of the muscular dystrophies. Expert Rev Mol Med (2009) 1.29
Beta 2-agonist administration reverses muscle wasting and improves muscle function in aged rats. J Physiol (2003) 1.25
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Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin. Neuromuscul Disord (2002) 1.24
Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol Ther (2010) 1.21
Transformation of madin-darby canine kidney epithelial cells by sheep retrovirus envelope proteins. J Virol (2005) 1.20
Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy. Hum Mol Genet (2008) 1.19
An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells. Nat Commun (2013) 1.17
Gene therapy strategies for Duchenne muscular dystrophy utilizing recombinant adeno-associated virus vectors. Mol Ther (2005) 1.15
Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin. J Cell Sci (2010) 1.14
Gene and cell-mediated therapies for muscular dystrophy. Muscle Nerve (2013) 1.14
Expression and characterization of a soluble, active form of the jaagsiekte sheep retrovirus receptor, Hyal2. J Virol (2005) 1.13
Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies. Mol Ther (2012) 1.13
Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature. Mol Ther (2009) 1.13
rAAV6-microdystrophin rescues aberrant Golgi complex organization in mdx skeletal muscles. Traffic (2007) 1.13
Elevated expression of activins promotes muscle wasting and cachexia. FASEB J (2013) 1.13
The bone morphogenetic protein axis is a positive regulator of skeletal muscle mass. J Cell Biol (2013) 1.12
Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging. Mol Ther (2009) 1.11
A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy. Hum Mol Genet (2006) 1.11
Interleukin-15 administration improves diaphragm muscle pathology and function in dystrophic mdx mice. Am J Pathol (2005) 1.10
Expression of Dp260 in muscle tethers the actin cytoskeleton to the dystrophin-glycoprotein complex and partially prevents dystrophy. Hum Mol Genet (2002) 1.10
Phenotypic improvement of dystrophic muscles by rAAV/microdystrophin vectors is augmented by Igf1 codelivery. Mol Ther (2005) 1.10