Published in Invest Ophthalmol Vis Sci on July 01, 2007
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther (2008) 2.93
Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency. Gene Ther (2010) 1.21
Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye. Mol Vis (2009) 1.16
AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa. Mol Ther (2011) 1.10
Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse. Exp Eye Res (2010) 1.07
Gene therapy for retinal disease. Transl Res (2013) 1.03
AAV retinal transduction in a large animal model species: comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector. Mol Vis (2009) 0.95
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Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus. Arch Ophthalmol (2010) 0.93
Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients. Hum Gene Ther (2012) 0.90
Corneal gene therapy: basic science and translational perspective. Ocul Surf (2013) 0.87
Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges. Expert Rev Hematol (2011) 0.87
Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4(-/-) mouse and bipolar cells in the rd1 mouse and human retina ex vivo. Gene Ther (2016) 0.78
Heparan Sulfate Binding Promotes Accumulation of Intravitreally Delivered Adeno-associated Viral Vectors at the Retina for Enhanced Transduction but Weakly Influences Tropism. J Virol (2016) 0.78
Self-Complementary Adeno-Associated Virus Vectors Improve Transduction Efficiency of Corneal Endothelial Cells. PLoS One (2016) 0.78
Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy. Hum Mol Genet (2015) 0.76
Identification of key residues determining isomerohydrolase activity of human RPE65. J Biol Chem (2014) 0.76
A TALE nuclease architecture for efficient genome editing. Nat Biotechnol (2010) 26.47
Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature (2005) 19.15
Genome editing with engineered zinc finger nucleases. Nat Rev Genet (2010) 18.88
An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol (2007) 17.80
Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol (2008) 14.75
Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases. Nat Biotechnol (2008) 14.20
Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol (2009) 12.40
Knockout rats via embryo microinjection of zinc-finger nucleases. Science (2009) 12.33
Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol (2011) 11.76
Targeted genome editing across species using ZFNs and TALENs. Science (2011) 9.84
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol (2007) 9.23
Distinct factors control histone variant H3.3 localization at specific genomic regions. Cell (2010) 8.79
Knockout rats generated by embryo microinjection of TALENs. Nat Biotechnol (2011) 8.45
Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med (2014) 7.64
Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases. Proc Natl Acad Sci U S A (2008) 6.59
Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell (2011) 6.56
Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol (2010) 6.55
An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44
Angiopoietin-2 is required for postnatal angiogenesis and lymphatic patterning, and only the latter role is rescued by Angiopoietin-1. Dev Cell (2002) 6.41
Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. Nat Methods (2010) 6.00
Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. Proc Natl Acad Sci U S A (2007) 5.93
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
Histone deimination antagonizes arginine methylation. Cell (2004) 5.19
In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature (2011) 4.81
Precise genome modification in the crop species Zea mays using zinc-finger nucleases. Nature (2009) 4.71
Ranibizumab for macular edema following central retinal vein occlusion: six-month primary end point results of a phase III study. Ophthalmology (2010) 4.60
Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther (2006) 4.36
Genetic variants near TIMP3 and high-density lipoprotein-associated loci influence susceptibility to age-related macular degeneration. Proc Natl Acad Sci U S A (2010) 4.30
Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med (2010) 4.28
Production and characterization of adeno-associated viral vectors. Nat Protoc (2006) 4.11
Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. Genome Res (2010) 3.94
Seven new loci associated with age-related macular degeneration. Nat Genet (2013) 3.81
Transient cold shock enhances zinc-finger nuclease-mediated gene disruption. Nat Methods (2010) 3.59
Two-year outcomes of the ranibizumab for edema of the mAcula in diabetes (READ-2) study. Ophthalmology (2010) 3.49
Cell type-specific regulation of angiogenic growth factor gene expression and induction of angiogenesis in nonischemic tissue by a constitutively active form of hypoxia-inducible factor 1. Circ Res (2003) 3.34
Investigation of the cause of death in a gene-therapy trial. N Engl J Med (2009) 3.02
Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther (2011) 2.91
Controlling long-range genomic interactions at a native locus by targeted tethering of a looping factor. Cell (2012) 2.87
Induction of angiogenesis in a mouse model using engineered transcription factors. Nat Med (2002) 2.81
Rapid and efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells. Nat Cell Biol (2011) 2.79
Primary End Point (Six Months) Results of the Ranibizumab for Edema of the mAcula in diabetes (READ-2) study. Ophthalmology (2009) 2.77
Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat Med (2003) 2.70
Sustained benefits from ranibizumab for macular edema following branch retinal vein occlusion: 12-month outcomes of a phase III study. Ophthalmology (2011) 2.65
Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol (2006) 2.63
Efficient generation of a biallelic knockout in pigs using zinc-finger nucleases. Proc Natl Acad Sci U S A (2011) 2.61
Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology. Nucleic Acids Res (2010) 2.61
Antioxidants reduce cone cell death in a model of retinitis pigmentosa. Proc Natl Acad Sci U S A (2006) 2.57
The AAV vector toolkit: poised at the clinical crossroads. Mol Ther (2012) 2.53
Vascular endothelial growth factor is a critical stimulus for diabetic macular edema. Am J Ophthalmol (2006) 2.50
Targeted transgene integration in plant cells using designed zinc finger nucleases. Plant Mol Biol (2008) 2.40
Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet (2004) 2.36
Ranibizumab for macular edema due to retinal vein occlusions: long-term follow-up in the HORIZON trial. Ophthalmology (2012) 2.36
Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods (2011) 2.30
Zinc-finger protein-targeted gene regulation: genomewide single-gene specificity. Proc Natl Acad Sci U S A (2003) 2.24
Generation of a triple-gene knockout mammalian cell line using engineered zinc-finger nucleases. Biotechnol Bioeng (2010) 2.23
DNA ligase III promotes alternative nonhomologous end-joining during chromosomal translocation formation. PLoS Genet (2011) 2.22
Remodeling of cortical bone allografts mediated by adherent rAAV-RANKL and VEGF gene therapy. Nat Med (2005) 2.10
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol (2009) 2.07
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol Ther (2011) 2.04
Pigment epithelium-derived factor suppresses ischemia-induced retinal neovascularization and VEGF-induced migration and growth. Invest Ophthalmol Vis Sci (2002) 2.03
Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme. Genome Res (2012) 2.02
Antioxidants slow photoreceptor cell death in mouse models of retinitis pigmentosa. J Cell Physiol (2007) 2.00
Gene-specific targeting of H3K9 methylation is sufficient for initiating repression in vivo. Curr Biol (2002) 1.90
Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Hum Gene Ther (2011) 1.90
Efficient targeted gene disruption in the soma and germ line of the frog Xenopus tropicalis using engineered zinc-finger nucleases. Proc Natl Acad Sci U S A (2011) 1.90
MicroRNAs regulate ocular neovascularization. Mol Ther (2008) 1.89
AAV hybrid serotypes: improved vectors for gene delivery. Curr Gene Ther (2005) 1.80
Site-specific genome editing in Plasmodium falciparum using engineered zinc-finger nucleases. Nat Methods (2012) 1.79
Intravitreal injection of bevacizumab for macular edema secondary to branch retinal vein occlusion:results after 12 months and multiple regression analysis. Retina (2009) 1.76
Translating dosage compensation to trisomy 21. Nature (2013) 1.75
Oxidative damage is a potential cause of cone cell death in retinitis pigmentosa. J Cell Physiol (2005) 1.73
Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX. Blood (2008) 1.72
VEGF-TRAP(R1R2) suppresses choroidal neovascularization and VEGF-induced breakdown of the blood-retinal barrier. J Cell Physiol (2003) 1.71
Efficient immunoglobulin gene disruption and targeted replacement in rabbit using zinc finger nucleases. PLoS One (2011) 1.69
Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J Virol (2004) 1.64
Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol Ther (2009) 1.61
Inducible expression of vascular endothelial growth factor in adult mice causes severe proliferative retinopathy and retinal detachment. Am J Pathol (2002) 1.59
An engineered zinc finger protein activator of the endogenous glial cell line-derived neurotrophic factor gene provides functional neuroprotection in a rat model of Parkinson's disease. J Neurosci (2010) 1.59
Targeted gene addition to human mesenchymal stromal cells as a cell-based plasma-soluble protein delivery platform. Cytotherapy (2010) 1.58
AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization. Invest Ophthalmol Vis Sci (2002) 1.58
Ranibizumab for edema of the macula in diabetes study: 3-year outcomes and the need for prolonged frequent treatment. JAMA Ophthalmol (2013) 1.55
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther (2013) 1.54
Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc Natl Acad Sci U S A (2009) 1.53
Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery. Hum Gene Ther (2011) 1.53
Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. J Virol (2007) 1.52
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Enhancement of adeno-associated virus infection by mobilizing capsids into and out of the nucleolus. J Virol (2008) 1.50
Angiopoietin-2 plays an important role in retinal angiogenesis. J Cell Physiol (2002) 1.50
Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases. PLoS Pathog (2011) 1.50
Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases. Mol Ther (2013) 1.49
The SDF-1/CXCR4 ligand/receptor pair is an important contributor to several types of ocular neovascularization. FASEB J (2007) 1.49
Ultrastructure of internal limiting membrane removed during plasmin-assisted vitrectomy from eyes with diabetic macular edema. Ophthalmology (2004) 1.48