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2005
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Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.
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2009
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2.72
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2003
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2.68
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In utero gene therapy rescues vision in a murine model of congenital blindness.
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2004
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10
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2008
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Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies.
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The human visual cortex responds to gene therapy-mediated recovery of retinal function.
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J Clin Invest
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Mutations in RPGR and RP2 account for 15% of males with simplex retinal degenerative disease.
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Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration.
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Mol Ther
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2004
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1.35
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Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness.
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Sci Transl Med
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1.35
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Disruption of intraflagellar protein transport in photoreceptor cilia causes Leber congenital amaurosis in humans and mice.
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Herpes simplex virus type 1 glycoprotein e is required for axonal localization of capsid, tegument, and membrane glycoproteins.
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1.29
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Tie2Cre-mediated inactivation of plexinD1 results in congenital heart, vascular and skeletal defects.
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Systemic rapamycin inhibits retinal and choroidal neovascularization in mice.
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Progress in gene therapy for neurological disorders.
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Dissecting the axoneme interactome: the mammalian orthologue of Chlamydomonas PF6 interacts with sperm-associated antigen 6, the mammalian orthologue of Chlamydomonas PF16.
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Mol Cell Proteomics
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Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa.
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In vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory function.
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Mol Ther
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Deficiency of SPAG16L causes male infertility associated with impaired sperm motility.
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Oxygen distribution and vascular injury in the mouse eye measured by phosphorescence-lifetime imaging.
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Increased expression of ceruloplasmin in the retina following photic injury.
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Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates.
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Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye.
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Mol Ther
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Accelerated mortality from hydrocephalus and pneumonia in mice with a combined deficiency of SPAG6 and SPAG16L reveals a functional interrelationship between the two central apparatus proteins.
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Mol Ther Nucleic Acids
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Persistent fetal ocular vasculature in mice deficient in bax and bak.
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Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor.
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A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus.
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Mol Ther
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Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transduction.
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1.02
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Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector.
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Mol Ther
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1.02
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Disruption of CEP290 microtubule/membrane-binding domains causes retinal degeneration.
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50
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AAV9 targets cone photoreceptors in the nonhuman primate retina.
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Macular pigment and lutein supplementation in choroideremia.
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Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients.
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Novel mutation in the TIMP3 gene causes Sorsby fundus dystrophy.
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Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents.
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Proapoptotic bcl-2 family members, Bax and Bak, are essential for developmental photoreceptor apoptosis.
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The distribution, concentration, and toxicity of enhanced green fluorescent protein in retinal cells after genomic or somatic (virus-mediated) gene transfer.
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