Published in Hum Gene Ther on December 12, 2010
Production and titering of recombinant adeno-associated viral vectors. J Vis Exp (2011) 1.41
Viral vectors for gene delivery to the central nervous system. Neurobiol Dis (2011) 1.20
Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma. Hum Gene Ther (2011) 1.13
Effective delivery of large genes to the retina by dual AAV vectors. EMBO Mol Med (2013) 1.04
Gene therapy for retinal disease. Transl Res (2013) 1.03
Oversized AAV transductifon is mediated via a DNA-PKcs-independent, Rad51C-dependent repair pathway. Mol Ther (2013) 1.02
Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy. Hum Mol Genet (2013) 0.99
Vector platforms for gene therapy of inherited retinopathies. Prog Retin Eye Res (2014) 0.94
Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery. Hum Gene Ther Methods (2014) 0.91
Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A. Hum Gene Ther Methods (2014) 0.90
Full-length dystrophin reconstitution with adeno-associated viral vectors. Hum Gene Ther (2014) 0.89
Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids. Hum Gene Ther Methods (2016) 0.83
A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence. Mol Ther Methods Clin Dev (2015) 0.83
Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons. Mol Brain (2014) 0.83
Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease. Hum Mol Genet (2015) 0.82
Gene Therapy of ABCA4-Associated Diseases. Cold Spring Harb Perspect Med (2015) 0.81
The production of viral vectors designed to express large and difficult to express transgenes within neurons. Mol Brain (2015) 0.79
Gene therapy to rescue retinal degeneration caused by mutations in rhodopsin. Methods Mol Biol (2015) 0.78
Transduction of skeletal muscles with common reporter genes can promote muscle fiber degeneration and inflammation. PLoS One (2012) 0.77
Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice. Mol Ther (2017) 0.77
Use of Adeno-Associated and Herpes Simplex Viral Vectors for In Vivo Neuronal Expression in Mice. Curr Protoc Neurosci (2015) 0.76
Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors. Methods Mol Biol (2016) 0.76
Copackaging of multiple adeno-associated viral vectors in a single production step. Hum Gene Ther Methods (2014) 0.76
Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy. Expert Opin Orphan Drugs (2015) 0.75
Preclinical Development of New Therapy for Glycogen Storage Diseases. Curr Gene Ther (2015) 0.75
Gene therapy approaches for prevention of retinal degeneration in Usher syndrome. Gene Ther (2017) 0.75
Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J Clin Invest (2000) 3.57
Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther (1996) 3.26
Effect of genome size on AAV vector packaging. Mol Ther (2009) 2.77
A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol Ther (2008) 2.22
Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol (2005) 2.16
Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps. J Virol (2005) 2.05
Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration. Gene Ther (2007) 2.01
Characterization of genome integrity for oversized recombinant AAV vector. Mol Ther (2009) 1.71
Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat Biotechnol (2002) 1.69
Isolation of two cell lines from a human malignant glioma specimen differing in sensitivity to radiation and chemotherapeutic drugs. Radiat Res (1993) 1.64
Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb. Mol Ther (2009) 1.64
Improved adeno-associated virus vector production with transfection of a single helper adenovirus gene, E4orf6. Mol Ther (2000) 1.55
Efficient whole-body transduction with trans-splicing adeno-associated viral vectors. Mol Ther (2007) 1.48
Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle. J Gene Med (2006) 1.36
A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner. Mol Ther (2007) 1.31
Expanding adeno-associated viral vector capacity: a tale of two vectors. Biotechnol Genet Eng Rev (2007) 1.06
Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med (2002) 5.07
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
Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation (2003) 2.22
A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol Ther (2008) 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
The passive mechanical properties of the extensor digitorum longus muscle are compromised in 2- to 20-mo-old mdx mice. J Appl Physiol (1985) (2011) 1.75
Prevention of dystrophin-deficient cardiomyopathy in twenty-one-month-old carrier mice by mosaic dystrophin expression or complementary dystrophin/utrophin expression. Circ Res (2007) 1.65
Evidence for impaired neurovascular transmission in a murine model of Duchenne muscular dystrophy. J Appl Physiol (1985) (2010) 1.64
Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb. Mol Ther (2009) 1.64
Systemic Trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for duchenne muscular dystrophy. Hum Gene Ther (2009) 1.53
Efficient whole-body transduction with trans-splicing adeno-associated viral vectors. Mol Ther (2007) 1.48
Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle. J Gene Med (2006) 1.36
Cardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice. Mol Ther (2008) 1.36
Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice. Hum Gene Ther (2008) 1.36
Preservation of muscle force in Mdx3cv mice correlates with low-level expression of a near full-length dystrophin protein. Am J Pathol (2008) 1.33
A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner. Mol Ther (2007) 1.31
Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle. Hum Gene Ther (2010) 1.26
Full-length dystrophin expression in half of the heart cells ameliorates beta-isoproterenol-induced cardiomyopathy in mdx mice. Hum Mol Genet (2004) 1.25
Monitoring murine skeletal muscle function for muscle gene therapy. Methods Mol Biol (2011) 1.22
Nitrosative stress elicited by nNOSµ delocalization inhibits muscle force in dystrophin-null mice. J Pathol (2010) 1.20
An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in Duchenne-like muscular dystrophy in the corgi breed. Lab Invest (2010) 1.20
Consequences of DNA-dependent protein kinase catalytic subunit deficiency on recombinant adeno-associated virus genome circularization and heterodimerization in muscle tissue. J Virol (2003) 1.16
Trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mRNA but not by dual vector coinfection efficiency. Hum Gene Ther (2004) 1.16
Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin. J Cell Sci (2010) 1.14
Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice. PLoS One (2010) 1.14
Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy. Mol Ther (2013) 1.13
Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma. Hum Gene Ther (2011) 1.13
Sub-physiological sarcoglycan expression contributes to compensatory muscle protection in mdx mice. Hum Mol Genet (2009) 1.12
α2 and α3 helices of dystrophin R16 and R17 frame a microdomain in the α1 helix of dystrophin R17 for neuronal NOS binding. Proc Natl Acad Sci U S A (2012) 1.11
AAV-microdystrophin therapy improves cardiac performance in aged female mdx mice. Mol Ther (2011) 1.08
Expanding adeno-associated viral vector capacity: a tale of two vectors. Biotechnol Genet Eng Rev (2007) 1.06
Age-matched comparison reveals early electrocardiography and echocardiography changes in dystrophin-deficient dogs. Neuromuscul Disord (2011) 1.04
Whole body skeletal muscle transduction in neonatal dogs with AAV-9. Methods Mol Biol (2011) 1.03
Genotyping mdx, mdx3cv, and mdx4cv mice by primer competition polymerase chain reaction. Muscle Nerve (2010) 1.03
A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs. Hum Gene Ther (2011) 1.00
AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy. J Mol Cell Cardiol (2012) 1.00
Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy. Hum Mol Genet (2013) 0.99
Gender influences cardiac function in the mdx model of Duchenne cardiomyopathy. Muscle Nerve (2010) 0.99
Double strand interaction is the predominant pathway for intermolecular recombination of adeno-associated viral genomes. Virology (2003) 0.99
Gender differences in contractile and passive properties of mdx extensor digitorum longus muscle. Muscle Nerve (2012) 0.98
Adeno-associated virus serotype-9 efficiently transduces the retinal outer plexiform layer. Mol Vis (2009) 0.97
Synthetic intron improves transduction efficiency of trans-splicing adeno-associated viral vectors. Hum Gene Ther (2006) 0.95
Recombinant AAV-mediated gene delivery using dual vector heterodimerization. Methods Enzymol (2002) 0.95
Ectopic catalase expression in mitochondria by adeno-associated virus enhances exercise performance in mice. PLoS One (2009) 0.95
Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model of Duchenne cardiomyopathy. Hum Mol Genet (2013) 0.94
Phenotyping cardiac gene therapy in mice. Methods Mol Biol (2011) 0.94
Long-term robust myocardial transduction of the dog heart from a peripheral vein by adeno-associated virus serotype-8. Hum Gene Ther (2013) 0.93
Evaluation of muscle function of the extensor digitorum longus muscle ex vivo and tibialis anterior muscle in situ in mice. J Vis Exp (2013) 0.93
Recombinant adeno-associated viral vector production and purification. Methods Mol Biol (2012) 0.92
The evolution of heart gene delivery vectors. J Gene Med (2011) 0.92
SERCA2a gene transfer improves electrocardiographic performance in aged mdx mice. J Transl Med (2011) 0.92
Dystrophin deficiency compromises force production of the extensor carpi ulnaris muscle in the canine model of Duchenne muscular dystrophy. PLoS One (2012) 0.90
Humoral immunity to AAV-6, 8, and 9 in normal and dystrophic dogs. Hum Gene Ther (2012) 0.89
Quantitative phenotyping of Duchenne muscular dystrophy dogs by comprehensive gait analysis and overnight activity monitoring. PLoS One (2013) 0.89
Dual vector expansion of the recombinant AAV packaging capacity. Methods Mol Biol (2003) 0.89
Full-length dystrophin reconstitution with adeno-associated viral vectors. Hum Gene Ther (2014) 0.89
Trans-splicing vectors expand the packaging limits of adeno-associated virus for gene therapy applications. Methods Mol Med (2003) 0.83
iNOS ablation does not improve specific force of the extensor digitorum longus muscle in dystrophin-deficient mdx4cv mice. PLoS One (2011) 0.83
Design of trans-splicing adeno-associated viral vectors for Duchenne muscular dystrophy gene therapy. Methods Mol Biol (2008) 0.82
Adenovirus-mediated gene transfer to adult mouse cardiomyocytes is selectively influenced by culture medium. J Gene Med (2003) 0.81
Adeno-associated virus serotype-9 mediated retinal outer plexiform layer transduction is mainly through the photoreceptors. Adv Exp Med Biol (2010) 0.81