Published in J Gene Med on June 01, 2003
Mitochondrial optic neuropathies - disease mechanisms and therapeutic strategies. Prog Retin Eye Res (2010) 3.58
Regulatable gene expression systems for gene therapy applications: progress and future challenges. Mol Ther (2005) 1.61
AAV-mediated local delivery of interferon-beta for the treatment of retinoblastoma in preclinical models. Neuromolecular Med (2009) 0.95
Regulatable gene expression systems for gene therapy. Curr Gene Ther (2006) 0.92
Lack of humoral immune response to the tetracycline (Tet) activator in rats injected intracranially with Tet-off rAAV vectors. Gene Ther (2010) 0.92
Gene therapy for ocular diseases. Br J Ophthalmol (2010) 0.90
Gene therapy targeting glaucoma: where are we? Surv Ophthalmol (2009) 0.83
Regulation of retinal function but nonrescue of vision in RPE65-deficient dogs treated with doxycycline-regulatable AAV vectors. Mol Ther (2010) 0.80
Long-term inducible expression in striatal neurons from helper virus-free HSV-1 vectors that contain the tetracycline-inducible promoter system. Brain Res (2006) 0.79
Transgene regulation using the tetracycline-inducible TetR-KRAB system after AAV-mediated gene transfer in rodents and nonhuman primates. PLoS One (2014) 0.79
Republished review: Gene therapy for ocular diseases. Postgrad Med J (2011) 0.77
Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J Virol (2002) 5.82
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther (2009) 3.33
Chikungunya disease in nonhuman primates involves long-term viral persistence in macrophages. J Clin Invest (2010) 2.92
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
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle. J Virol (2008) 1.44
Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain. Mol Ther (2008) 1.41
A nonionic amphiphile agent promotes gene delivery in vivo to skeletal and cardiac muscles. Hum Gene Ther (2002) 1.37
Gene therapy of the brain in the dog model of Hurler's syndrome. Ann Neurol (2006) 1.32
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. Sci Transl Med (2014) 1.32
A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8. Mol Ther (2011) 1.29
Long-term doxycycline-regulated transgene expression in the retina of nonhuman primates following subretinal injection of recombinant AAV vectors. Mol Ther (2006) 1.24
In vivo gene regulation using tetracycline-regulatable systems. Adv Drug Deliv Rev (2009) 1.23
Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes. Mol Ther (2010) 1.23
Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle. Mol Ther (2009) 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
Texture analysis of magnetic resonance images of rat muscles during atrophy and regeneration. Magn Reson Imaging (2005) 1.17
Autoimmune anemia in macaques following erythropoietin gene therapy. Blood (2004) 1.16
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
Spontaneous abrogation of the G₂DNA damage checkpoint has clinical benefits but promotes leukemogenesis in Fanconi anemia patients. J Clin Invest (2010) 1.13
Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery. Mol Ther (2005) 1.10
Optimal design of a single recombinant adeno-associated virus derived from serotypes 1 and 2 to achieve more tightly regulated transgene expression from nonhuman primate muscle. Mol Ther (2004) 1.09
AAV-mediated gene therapy for retinal disorders in large animal models. ILAR J (2009) 1.08
Biodistribution of rAAV vectors following intraocular administration: evidence for the presence and persistence of vector DNA in the optic nerve and in the brain. Mol Ther (2005) 1.07
Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution. Hum Gene Ther (2009) 1.06
Postsurgical assessment and long-term safety of recombinant adeno-associated virus-mediated gene transfer into the retinas of dogs and primates. Arch Ophthalmol (2005) 1.02
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate. Hum Mol Genet (2010) 1.01
Restoration of vision in the pde6β-deficient dog, a large animal model of rod-cone dystrophy. Mol Ther (2012) 1.00
International efforts for recombinant adeno-associated viral vector reference standards. Mol Ther (2008) 1.00
Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6. J Virol (2012) 0.99
Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver. Mol Ther (2012) 0.98
The cellular TATA binding protein is required for rep-dependent replication of a minimal adeno-associated virus type 2 p5 element. J Virol (2005) 0.98
Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors. J Immunol (2012) 0.98
scAAV9 intracisternal delivery results in efficient gene transfer to the central nervous system of a feline model of motor neuron disease. Hum Gene Ther (2013) 0.98
Tetracycline-inducible viral interleukin-10 intraocular gene transfer, using adeno-associated virus in experimental autoimmune uveoretinitis. Hum Gene Ther (2005) 0.97
Muscle satellite cell heterogeneity: in vitro and in vivo evidences for populations that fuse differently. Cell Tissue Res (2004) 0.97
Photodynamic therapy of multiple nonmelanoma skin cancers with verteporfin and red light-emitting diodes: two-year results evaluating tumor response and cosmetic outcomes. Arch Dermatol (2004) 0.96
Progenitor cell isolation from muscle-derived cells based on adhesion properties. J Histochem Cytochem (2007) 0.95
Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat. Mol Ther (2002) 0.93
The RPGRIP1-deficient dog, a promising canine model for gene therapy. Mol Vis (2009) 0.92
"Genetic Doping" with erythropoietin cDNA in primate muscle is detectable. Mol Ther (2004) 0.92
Immune responses to gene product of inducible promoters. Curr Gene Ther (2007) 0.92
Herpes simplex virus type 1 ICP0 protein mediates activation of adeno-associated virus type 2 rep gene expression from a latent integrated form. J Virol (2004) 0.92
Epicutaneous immunotherapy (EPIT) blocks the allergic esophago-gastro-enteropathy induced by sustained oral exposure to peanuts in sensitized mice. PLoS One (2012) 0.92
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material. Hum Gene Ther (2014) 0.91
Identification of phenotypic discriminating markers for intervertebral disc cells and articular chondrocytes. Rheumatology (Oxford) (2009) 0.91
Adeno-associated viral vectors for retinal gene transfer and treatment of retinal diseases. Curr Gene Ther (2005) 0.90
Adeno-associated virus mediated gene therapy for retinal degenerative diseases. Methods Mol Biol (2011) 0.90
Superiority of bone marrow-derived dendritic cells over monocyte-derived ones for the expansion of regulatory T cells in the macaque. Transplantation (2008) 0.90
PTEN contributes to profound PI3K/Akt signaling pathway deregulation in dystrophin-deficient dog muscle. Am J Pathol (2009) 0.89
Cytotoxic immune response blunts long-term transgene expression after efficient retroviral-mediated hepatic gene transfer in rat. Mol Ther (2002) 0.87
The Fanconi anemia pathway and the DNA interstrand cross-links repair. Biochimie (2003) 0.87
Characterization of the age-dependent intervertebral disc changes in rabbit by correlation between MRI, histology and gene expression. BMC Musculoskelet Disord (2011) 0.85
Improving rAAV production and purification: towards the definition of a scaleable process. J Gene Med (2004) 0.84
Microvessel density in muscles of dogs with golden retriever muscular dystrophy. Neuromuscul Disord (2005) 0.84
Early detection of a two-long-terminal-repeat junction molecule in the cytoplasm of recombinant murine leukemia virus-infected cells. J Virol (2004) 0.84
Exacerbation of undiagnosed mycosis fungoides during treatment with etanercept. Arch Dermatol (2009) 0.83
Characterization of producer cell-dependent restriction of murine leukemia virus replication. J Virol (2002) 0.82
Adeno-associated viral vector-mediated transgene expression is independent of DNA methylation in primate liver and skeletal muscle. PLoS One (2011) 0.82
Gene therapeutic prospects in early onset of severe retinal dystrophy: restoration of vision in RPE65 Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium. Bull Mem Acad R Med Belg (2006) 0.82