Published in Invest Ophthalmol Vis Sci on January 01, 2007
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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
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Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther (2009) 3.07
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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
Natural variation in GS5 plays an important role in regulating grain size and yield in rice. Nat Genet (2011) 2.50
Dicer inactivation leads to progressive functional and structural degeneration of the mouse retina. J Neurosci (2008) 2.50
Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol Ther (2005) 2.47
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
Rescue of a deficiency in ATP synthesis by transfer of MTATP6, a mitochondrial DNA-encoded gene, to the nucleus. Nat Genet (2002) 2.20
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
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Learning brain connectivity of Alzheimer's disease by sparse inverse covariance estimation. Neuroimage (2010) 2.04
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
Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness. Mol Ther (2011) 1.91
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
Integrated profiling of microRNAs and mRNAs: microRNAs located on Xq27.3 associate with clear cell renal cell carcinoma. PLoS One (2010) 1.82
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther (2006) 1.81
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
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
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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
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
TrkB gene transfer protects retinal ganglion cells from axotomy-induced death in vivo. J Neurosci (2002) 1.51
Automated annotation of Drosophila gene expression patterns using a controlled vocabulary. Bioinformatics (2008) 1.50
A novel gene variation of TNFalpha associated with ankylosing spondylitis: a reconfirmed study. Ann Rheum Dis (2007) 1.48
A genome-wide association study identifies GRK5 and RASGRP1 as type 2 diabetes loci in Chinese Hans. Diabetes (2012) 1.47
Adeno-associated virus-vectored gene therapy for retinal disease. Hum Gene Ther (2005) 1.43
IL-10 suppresses chemokines, inflammation, and fibrosis in a model of chronic renal disease. J Am Soc Nephrol (2005) 1.41
Suppression of mitochondrial oxidative stress provides long-term neuroprotection in experimental optic neuritis. Invest Ophthalmol Vis Sci (2007) 1.40
Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1. Invest Ophthalmol Vis Sci (2006) 1.39
Functional genomic screening identifies dual leucine zipper kinase as a key mediator of retinal ganglion cell death. Proc Natl Acad Sci U S A (2013) 1.36
Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. PLoS One (2013) 1.33
Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS One (2010) 1.33
Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel. Hum Gene Ther (2010) 1.33
ACE2 and Ang-(1-7) confer protection against development of diabetic retinopathy. Mol Ther (2011) 1.32
Effect of CNTF on retinal ganglion cell survival in experimental glaucoma. Invest Ophthalmol Vis Sci (2008) 1.31
ER stress is involved in T17M rhodopsin-induced retinal degeneration. Invest Ophthalmol Vis Sci (2012) 1.31
Design, synthesis, and biological evaluation of new-generation taxoids. J Med Chem (2008) 1.29
AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia. PLoS One (2012) 1.28
Increased sensitivity to light-induced damage in a mouse model of autosomal dominant retinal disease. Invest Ophthalmol Vis Sci (2007) 1.27
[Ovarian steroid cell tumor, not otherwise specified: a clinicopathologic study]. Zhonghua Bing Li Xue Za Zhi (2007) 1.27
Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model. Mol Ther (2002) 1.26
In vivo RNAi-mediated alpha-synuclein silencing induces nigrostriatal degeneration. Mol Ther (2010) 1.26
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
Mitochondrial protein nitration primes neurodegeneration in experimental autoimmune encephalomyelitis. J Biol Chem (2006) 1.25
Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice. Hum Gene Ther (2012) 1.24
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
Glucose regulated protein 78 diminishes α-synuclein neurotoxicity in a rat model of Parkinson disease. Mol Ther (2012) 1.24
Use of the tubulin bound paclitaxel conformation for structure-based rational drug design. Chem Biol (2005) 1.24
Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathway. Proc Natl Acad Sci U S A (2005) 1.22
Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model. Proc Natl Acad Sci U S A (2011) 1.22
Molecular and cellular alterations induced by sustained expression of ciliary neurotrophic factor in a mouse model of retinitis pigmentosa. Invest Ophthalmol Vis Sci (2007) 1.22
AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa. Hum Gene Ther (2011) 1.20