Published in Mol Vis on February 06, 2009
Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy. Prog Retin Eye Res (2010) 1.81
Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS One (2010) 1.33
rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters. Gene Ther (2010) 1.22
Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency. Gene Ther (2010) 1.21
The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. Hum Gene Ther (2012) 1.16
Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis. Invest Ophthalmol Vis Sci (2011) 1.16
Gene therapy in the second eye of RPE65-deficient dogs improves retinal function. Gene Ther (2010) 1.09
Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse. Exp Eye Res (2010) 1.07
Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290. Mol Ther Nucleic Acids (2012) 1.05
Direct gene transfer with compacted DNA nanoparticles in retinal pigment epithelial cells: expression, repeat delivery and lack of toxicity. Nanomedicine (Lond) (2012) 0.93
AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice. Invest Ophthalmol Vis Sci (2014) 0.89
AAV-mediated gene therapy in mouse models of recessive retinal degeneration. Curr Mol Med (2012) 0.84
Differential proteomics and functional research following gene therapy in a mouse model of Leber congenital amaurosis. PLoS One (2012) 0.82
The Rpe65 rd12 allele exerts a semidominant negative effect on vision in mice. Invest Ophthalmol Vis Sci (2014) 0.81
Gene therapy for cardiovascular disease mediated by ultrasound and microbubbles. Cardiovasc Ultrasound (2013) 0.80
Trans-Corneal Subretinal Injection in Mice and Its Effect on the Function and Morphology of the Retina. PLoS One (2015) 0.76
The frequency-response electroretinogram distinguishes cone and abnormal rod function in rd12 mice. PLoS One (2015) 0.76
Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease. J Vis Exp (2015) 0.76
iTRAQ-Based Proteomic Analysis of Visual Cycle-Associated Proteins in RPE of rd12 Mice before and after RPE65 Gene Delivery. J Ophthalmol (2015) 0.75
Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials. Front Neurosci (2017) 0.75
Local and systemic responses following intravitreous injection of AAV2-encoded modified Volvox channelrhodopsin-1 in a genetically blind rat model. Gene Ther (2015) 0.75
The Degeneration and Apoptosis Patterns of Cone Photoreceptors in rd11 Mice. J Ophthalmol (2017) 0.75
Effects of Subretinal Gene Transfer at Different Time Points in a Mouse Model of Retinal Degeneration. PLoS One (2016) 0.75
Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med (2008) 17.21
Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med (2008) 15.30
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther (2008) 8.83
Gene therapy restores vision in a canine model of childhood blindness. Nat Genet (2001) 7.88
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A (2008) 7.16
Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol (1989) 6.69
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol (1996) 6.43
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther (2003) 4.92
Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci U S A (1996) 4.76
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med (2001) 4.02
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther (2008) 3.28
Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration. J Virol (1997) 2.65
Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA). Mol Vis (2005) 2.55
Characterization of the DNA of a defective human parvovirus isolated from a genital site. Virology (1984) 2.49
Restoration of cone vision in a mouse model of achromatopsia. Nat Med (2007) 2.44
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis. Mol Ther (2005) 2.27
Analysis of proteins, helper dependence, and seroepidemiology of a new human parvovirus. Virology (1984) 2.13
Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposure. J Virol (1998) 2.11
Epidemiology of adenovirus-associated virus infection in a nursery population. Am J Epidemiol (1968) 1.96
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci U S A (1999) 1.89
Transient immunosuppression allows transgene expression following readministration of adeno-associated viral vectors. Hum Gene Ther (1998) 1.79
Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size. J Virol (2002) 1.77
AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation. Invest Ophthalmol Vis Sci (2008) 1.68
Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Ther (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
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential. Mol Vis (2008) 1.59
Identification of the RPE65 protein in mammalian cone photoreceptors. Invest Ophthalmol Vis Sci (2002) 1.57
The role of FasL-induced apoptosis in immune privilege. Immunol Today (1997) 1.39
Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors. Mol Ther (2000) 1.32
Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis. Mol Vis (2007) 1.26
Immune regulation and the eye: a dangerous compromise. FASEB J (1987) 1.25
Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: effects of serotype and site of administration. Vision Res (2007) 1.24
Analysis of immune deviation elicited by antigens injected into the subretinal space. Invest Ophthalmol Vis Sci (1998) 1.05
Ocular gene transfer with self-complementary AAV vectors. Invest Ophthalmol Vis Sci (2007) 1.04
A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus. Mol Ther (2002) 1.04
Additional transduction events after subretinal readministration of recombinant adeno-associated virus. Hum Gene Ther (2000) 1.02
Human retinal pigment epithelial cell-induced apoptosis in activated T cells. Invest Ophthalmol Vis Sci (1998) 1.00
Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function. Vision Res (2006) 0.99
Cross-species comparison of in vivo reporter gene expression after recombinant adeno-associated virus-mediated retinal transduction. Methods Enzymol (2000) 0.94
Role of Th1 and Th2 cells in anterior chamber-associated immune deviation. Immunology (1996) 0.93
Production of latent transforming growth factor-beta and other inhibitory factors by cultured murine iris and ciliary body cells. Curr Eye Res (1991) 0.90
Analysis of immunosuppressive properties of iris and ciliary body cells and their secretory products. Invest Ophthalmol Vis Sci (1991) 0.90
Unconventional rejection of neural retinal allografts implanted into the immunologically privileged site of the eye. Transplantation (1995) 0.89
Ribozyme gene therapy for autosomal dominant retinal disease. Clin Chem Lab Med (2000) 0.85
The Gordon Wilson Lecture. From basic virology to human gene therapy. Trans Am Clin Climatol Assoc (1999) 0.81
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A (2008) 7.16
A muscleblind knockout model for myotonic dystrophy. Science (2003) 5.93
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods (2002) 5.30
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther (2005) 4.74
APD2: the updated antimicrobial peptide database and its application in peptide design. Nucleic Acids Res (2008) 4.23
DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration. Nature (2011) 3.90
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol (2011) 3.79
Pivotal role of dermal IL-17-producing γδ T cells in skin inflammation. Immunity (2011) 3.75
Meta-analysis of genome-wide association studies of asthma in ethnically diverse North American populations. Nat Genet (2011) 3.68
Mutations in genes encoding melanosomal proteins cause pigmentary glaucoma in DBA/2J mice. Nat Genet (2001) 3.30
A high-resolution map of segmental DNA copy number variation in the mouse genome. PLoS Genet (2006) 3.24
Improvement and decline in vision with gene therapy in childhood blindness. N Engl J Med (2015) 3.16
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther (2009) 3.07
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther (2008) 2.93
China: the aging giant. J Am Geriatr Soc (2007) 2.89
DICER1 loss and Alu RNA induce age-related macular degeneration via the NLRP3 inflammasome and MyD88. Cell (2012) 2.81
CRB1 is essential for external limiting membrane integrity and photoreceptor morphogenesis in the mammalian retina. Hum Mol Genet (2003) 2.77
Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci U S A (2013) 2.71
Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med (2009) 2.57
Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA). Mol Vis (2005) 2.55
Dicer inactivation leads to progressive functional and structural degeneration of the mouse retina. J Neurosci (2008) 2.50
The role of pyocyanin in Pseudomonas aeruginosa infection. Trends Mol Med (2004) 2.48
Restoration of cone vision in a mouse model of achromatopsia. Nat Med (2007) 2.44
Gene therapy for red-green colour blindness in adult primates. Nature (2009) 2.44
Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78. Proc Natl Acad Sci U S A (2010) 2.40
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis. Mol Ther (2005) 2.27
MicroRNA-1/206 targets c-Met and inhibits rhabdomyosarcoma development. J Biol Chem (2009) 2.24
Gene therapy rescues cone function in congenital achromatopsia. Hum Mol Genet (2010) 2.18
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol Ther (2006) 2.13
Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy. Ann Neurol (2002) 2.13
Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Proc Natl Acad Sci U S A (2012) 2.12
Pseudomonas aeruginosa pyocyanin is critical for lung infection in mice. Infect Immun (2004) 2.03
Towards precise classification of cancers based on robust gene functional expression profiles. BMC Bioinformatics (2005) 2.01
Human immunodeficiency virus type 1 enters brain microvascular endothelia by macropinocytosis dependent on lipid rafts and the mitogen-activated protein kinase signaling pathway. J Virol (2002) 1.97
Brain microglial cytokines in neurogenic hypertension. Hypertension (2010) 1.97
MiRNA-miRNA synergistic network: construction via co-regulating functional modules and disease miRNA topological features. Nucleic Acids Res (2010) 1.94
Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model. Proc Natl Acad Sci U S A (2012) 1.94
Transmission of MDR and XDR tuberculosis in Shanghai, China. PLoS One (2009) 1.93
Meta-analysis of genome-wide association studies from the CHARGE consortium identifies common variants associated with carotid intima media thickness and plaque. Nat Genet (2011) 1.92
Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness. Mol Ther (2011) 1.91
Brain cannabinoid CB₂ receptors modulate cocaine's actions in mice. Nat Neurosci (2011) 1.88
Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model. Invest Ophthalmol Vis Sci (2003) 1.83
Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis. PLoS Med (2005) 1.83
Mouse models of USH1C and DFNB18: phenotypic and molecular analyses of two new spontaneous mutations of the Ush1c gene. Hum Mol Genet (2003) 1.83
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther (2006) 1.81
Major taste loss in carnivorous mammals. Proc Natl Acad Sci U S A (2012) 1.81
MicroRNA-34a inhibits uveal melanoma cell proliferation and migration through downregulation of c-Met. Invest Ophthalmol Vis Sci (2008) 1.80
Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis. Mol Ther (2005) 1.79
Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation. PLoS Med (2007) 1.79
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Mol Ther (2010) 1.77
SOD2 knockdown mouse model of early AMD. Invest Ophthalmol Vis Sci (2007) 1.77
The tumorigenic FGFR3-TACC3 gene fusion escapes miR-99a regulation in glioblastoma. J Clin Invest (2013) 1.76
Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa. Mol Ther (2010) 1.75
trans-Diaqua-bis[5-carb-oxy-4-carboxyl-ato-2-(4-pyridinio)-1H-imidazol-1-ido-κN,O]iron(II). Acta Crystallogr Sect E Struct Rep Online (2009) 1.72
A comprehensive review of retinal gene therapy. Mol Ther (2013) 1.70
SOD2 gene transfer protects against optic neuropathy induced by deficiency of complex I. Ann Neurol (2004) 1.70
AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation. Invest Ophthalmol Vis Sci (2008) 1.68
Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis. PLoS Med (2006) 1.67
Centrosomal-ciliary gene CEP290/NPHP6 mutations result in blindness with unexpected sparing of photoreceptors and visual brain: implications for therapy of Leber congenital amaurosis. Hum Mutat (2007) 1.67
Correlation of iron in the hippocampus with MMSE in patients with Alzheimer's disease. J Magn Reson Imaging (2009) 1.64
Intraocular and systemic pharmacokinetics of triamcinolone acetonide after a single 40-mg posterior subtenon application. Ophthalmology (2010) 1.64
Premature truncation of a novel protein, RD3, exhibiting subnuclear localization is associated with retinal degeneration. Am J Hum Genet (2006) 1.63
Mfrp, a gene encoding a frizzled related protein, is mutated in the mouse retinal degeneration 6. Hum Mol Genet (2002) 1.63
Droplet networks with incorporated protein diodes show collective properties. Nat Nanotechnol (2009) 1.61
Digenic inheritance of deafness caused by mutations in genes encoding cadherin 23 and protocadherin 15 in mice and humans. Hum Mol Genet (2004) 1.61
High-efficiency and heritable gene targeting in mouse by transcription activator-like effector nucleases. Nucleic Acids Res (2013) 1.61
Moderate cardiac-selective overexpression of angiotensin II type 2 receptor protects cardiac functions from ischaemic injury. Exp Physiol (2011) 1.61
The Highwire ubiquitin ligase promotes axonal degeneration by tuning levels of Nmnat protein. PLoS Biol (2012) 1.60
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential. Mol Vis (2008) 1.59
AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization. Invest Ophthalmol Vis Sci (2002) 1.58
Structures of ABCB10, a human ATP-binding cassette transporter in apo- and nucleotide-bound states. Proc Natl Acad Sci U S A (2013) 1.54
Effect of transcutaneous auricular vagus nerve stimulation on impaired glucose tolerance: a pilot randomized study. BMC Complement Altern Med (2014) 1.54
Uptake, recycling, and antioxidant actions of alpha-lipoic acid in endothelial cells. Free Radic Biol Med (2002) 1.54
Allogeneic mesenchymal stem cell transplantation in severe and refractory systemic lupus erythematosus: 4 years of experience. Cell Transplant (2013) 1.53
Deletion of G protein-coupled receptor 48 leads to ocular anterior segment dysgenesis (ASD) through down-regulation of Pitx2. Proc Natl Acad Sci U S A (2008) 1.53
Mouse model of subretinal neovascularization with choroidal anastomosis. Retina (2003) 1.52
TrkB gene transfer protects retinal ganglion cells from axotomy-induced death in vivo. J Neurosci (2002) 1.51
Mouse models of age-related macular degeneration. Exp Eye Res (2005) 1.49
Apparently low reproducibility of true differential expression discoveries in microarray studies. Bioinformatics (2008) 1.48