Citation Impact
Citing Papers
DNA capture by a CRISPR-Cas9–guided adenine base editor
2020 StandoutScienceNobel
Inhibition of vascular endothelial growth factor with a sequence-specific hypoxia response element antagonist
2004 StandoutNobel
Formylglycine-generating enzyme binds substrate directly at a mononuclear Cu(I) center to initiate O 2 activation
2019 StandoutNobel
NEURONAL CIRCUITS OF THE NEOCORTEX
2004
Identification of the RPE65 protein in mammalian cone photoreceptors.
2002
Therapeutic siRNA: state of the art
2020
Hallmarks of Cancer: The Next Generation
2011 Standout
RPGR-ORF15, Which Is Mutated in Retinitis Pigmentosa, Associates with SMC1, SMC3, and Microtubule Transport Proteins
2005
Canine and Human Visual Cortex Intact and Responsive Despite Early Retinal Blindness from RPE65 Mutation
2007
The promise and challenge of therapeutic genome editing
2020 StandoutNatureNobel
Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
2008 Standout
Crystal Structure of the HSV-1 Fc Receptor Bound to Fc Reveals a Mechanism for Antibody Bipolar Bridging
2006 StandoutNobel
Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors
2005
Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium
2006
The Retinal Pigment Epithelium in Visual Function
2005 Standout
Inflammation and metabolic disorders
2006 StandoutNature
Axon Guidance Molecules in Vascular Patterning
2010
A Novel Locus (RP24) for X-linked Retinitis Pigmentosa Maps to Xq26-27
1998
Vision Change After Sheet Transplant of Fetal Retina With Retinal PigmentEpithelium to a Patient With Retinitis Pigmentosa
2004
Encapsidated adenovirus mini-chromosome-mediated delivery of genes to the retina: application to the rescue of photoreceptor degeneration
1998
Photochemical Restoration of Visual Responses in Blind Mice
2012
Immune responses to gene therapy vectors: influence on vector function and effector mechanisms
2004
Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial
2015
Pro-resolving lipid mediators are leads for resolution physiology
2014 StandoutNature
In Vivo Gene Therapy in Young and Adult RPE65-/- Dogs Produces Long-Term Visual Improvement
2003
New views on RPE65 deficiency: the rod system is the source of vision in a mouse model of Leber congenital amaurosis
2001
A Functional Rhodopsin-Green Fluorescent Protein Fusion Protein Localizes Correctly in Transgenic Xenopus laevis Retinal Rods and Is Expressed in a Time-dependent Pattern
2001
State-of-the-art gene-based therapies: the road ahead
2011
Transgenic mice with a rhodopsin mutation (Pro23His): A mouse model of autosomal dominant retinitis pigmentosa
1992
Degeneration of skeletal muscle, peripheral nerves, and the central nervous system in transgenic mice overexpressing wild-type prion proteins
1994 StandoutNobel
Pigment epithelium-derived factor in the monkey retinal pigment epithelium and interphotoreceptor matrix: apical secretion and distribution
2003
The Multiple Sulfatase Deficiency Gene Encodes an Essential and Limiting Factor for the Activity of Sulfatases
2003
Tissue-specific and developmental regulation of rod opsin chimeric genes in transgenic mice
1991
Update on the molecular genetics of retinitis pigmentosa
2001
Compound subretinal prostheses with extra-ocular parts designed for human trials: successful long-term implantation in pigs
2006
Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle
1998
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
2018
Crx, a Novel otx-like Homeobox Gene, Shows Photoreceptor-Specific Expression and Regulates Photoreceptor Differentiation
1997
Longitudinal and cross-sectional study of patients with early-onset severe retinal dystrophy associated with RPE65 mutations
2004
Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses
2020
NEW EMBO MEMBER'S REVIEW: On the genetics of retinitis pigmentosa and on mutation-independent approaches to therapeutic intervention
2002
Anatomical Profiling of Nuclear Receptor Expression Reveals a Hierarchical Transcriptional Network
2006
The Association between Visual Acuity and Central Retinal Thickness in Retinitis Pigmentosa
2005
RP2 and RPGR Mutations and Clinical Correlations in Patients with X-Linked Retinitis Pigmentosa
2003
Ephrin-B2 regulates VEGFR2 function in developmental and tumour angiogenesis
2010 Nature
Dark adaptation and the retinoid cycle of vision
2004
Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy
1996
Microvascular Modifications in Diabetic Retinopathy
2011
Retinitis pigmentosa
2006 Standout
Shared Mutations in NR2E3 in Enhanced S-cone Syndrome, Goldmann-Favre Syndrome, and Many Cases of Clumped Pigmentary Retinal Degeneration
2003
Hydration and DNA Recognition by Homeodomains
1996 StandoutNobel
The structure and function of G-protein-coupled receptors
2009 StandoutNatureNobel
In vivo genome editing using Staphylococcus aureus Cas9
2015 StandoutNature
Precise hit: adeno-associated virus in gene targeting
2005
Therapeutic modulation of endogenous gene function by agents with designed DNA-sequence specificities
2003
Gene transfer in the RPE65 null mutation dog: relationship between construct volume, visual behavior and electroretinographic (ERG) results
2003
A locus control region adjacent to the human red and green visual pigment genes
1992
Sequence analysis of the 5.34-kb 5′ flanking region of the human rhodopsin-encoding gene
1995 StandoutNobel
Age-related macular degeneration
2018 Standout
Computational redesign of endonuclease DNA binding and cleavage specificity
2006 StandoutNatureNobel
Conformational control of DNA target cleavage by CRISPR–Cas9
2015 StandoutNatureNobel
Crx, a Novel Otx-like Paired-Homeodomain Protein, Binds to and Transactivates Photoreceptor Cell-Specific Genes
1997
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
2011
What we can do and what we cannot do with fMRI
2008 StandoutNature
Genetic Defects in Vitamin A Metabolism of the Retinal Pigment Epithelium
2003
Retinal and choroidal angiogenesis: pathophysiology and strategies for inhibition
2003
Biology and Applications of CRISPR Systems: Harnessing Nature’s Toolbox for Genome Engineering
2016 StandoutNobel
Revealing the world of RNA interference
2004 StandoutNatureNobel
Assessment of Structure and Function Over a 3-year Period after Gene Transfer in RPE65−/− dogs
2005
Cerebral organoids model human brain development and microcephaly
2013 StandoutNature
Vitamin A metabolism in the retinal pigment epithelium: genes, mutations, and diseases
2003
A randomized, placebo-controlled clinical trial of docosahexaenoic acid supplementation for X-linked retinitis pigmentosa
2004
Allelic inactivation regulates olfactory receptor gene expression
1994 StandoutNobel
Probing the Functional Equivalence of Otoferlin and Synaptotagmin 1 in Exocytosis
2011 StandoutNobel
Essential functions of synapsins I and II in synaptic vesicle regulation
1995 StandoutNatureNobel
Molecular mechanisms and clinical applications of angiogenesis
2011 StandoutNature
Rescue and repair during photoreceptor cell renewal mediated by docosahexaenoic acid-derived neuroprotectin D1
2010
Angiogenesis as a therapeutic target
2005 StandoutNature
High resolution crystal structure of a paired (Pax) class cooperative homeodomain dimer on DNA
1995
Fluorescent indicators for Ca2+based on green fluorescent proteins and calmodulin
1997 StandoutNatureNobel
High-Titer Adeno-Associated Viral Vectors from a Rep/Cap Cell Line and Hybrid Shuttle Virus
1998
Gene Therapy in Neovascular Age-related Macular Degeneration: Three-Year Follow-up of a Phase 1 Randomized Dose Escalation Trial
2017
Delivery and Specificity of CRISPR/Cas9 Genome Editing Technologies for Human Gene Therapy
2015
Endogenous Inhibitors of Angiogenesis
2005
Activity of the Caenorhabditis elegans UNC-86 POU transcription factor modulates olfactory sensitivity
2003 StandoutNobel
Rescue from Photoreceptor Degeneration in the rd Mouse by Human Immunodeficiency Virus Vector-Mediated Gene Transfer
1999
Sulfatase-activated fluorophores for rapid discrimination of mycobacterial species and strains
2013 StandoutNobel
The human AQP4 gene: definition of the locus encoding two water channel polypeptides in brain.
1996 StandoutNobel
Angiopoietin-like 4 is a potent angiogenic factor and a novel therapeutic target for patients with proliferative diabetic retinopathy
2015 StandoutNobel
Clinical features and mutations in patients with dominant retinitis pigmentosa-1 (RP1).
2001
Targeted and Equally Distributed Delivery of mRNA to Organs with Pentaerythritol-Based One-Component Ionizable Amphiphilic Janus Dendrimers
2023 StandoutNobel
In vivo genome editing of the albumin locus as a platform for protein replacement therapy
2015
THE GREEN FLUORESCENT PROTEIN
1998 StandoutNobel
Aquaporin-1 Channels in Human Retinal Pigment Epithelium: Role in Transepithelial Water Movement
2003
One-Component Multifunctional Sequence-Defined Ionizable Amphiphilic Janus Dendrimer Delivery Systems for mRNA
2021 StandoutNobel
A million spiking-neuron integrated circuit with a scalable communication network and interface
2014 StandoutScience
Rescue of retinal degeneration by intravitreally injected adult bone marrow–derived lineage-negative hematopoietic stem cells
2004
Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk
2001
The Proximal Promoter of the Mouse Arrestin Gene Directs Gene Expression in Photoreceptor Cells and Contains an Evolutionarily Conserved Retinal Factor-Binding Site
1993
Intraocular adenoviral vector-mediated gene transfer in proliferative retinopathies.
2002
Electroporation and RNA interference in the rodent retina in vivo and in vitro
2003
Functional redundancy of cryptochromes and classical photoreceptors for nonvisual ocular photoreception in mice
2000 StandoutNobel
Adenoviral and AAV-Mediated Gene Transfer to the Inner Ear: Role of Serotype, Promoter, and Viral Load on In Vivo and In Vitro Infection Efficiencies
2009
Neural Science
2000 StandoutNobel
Reversible Photocontrol of Biological Systems by the Incorporation of Molecular Photoswitches
2013 StandoutNobel
Evidence for a new locus for X-linked retinitis pigmentosa (RP23).
2000
A novel domain suggests a ciliary function for ASPM, a brain size determining gene
2006
Science
2023 StandoutScience
A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells
1996
Recessive NRL mutations in patients with clumped pigmentary retinal degeneration and relative preservation of blue cone function
2004
BRAIN WORK AND BRAIN IMAGING
2006
Molecular Genetics of Human Retinal Disease
1999
Deuterium Relaxation in a Uniformly 15N-Labeled Homeodomain and Its DNA Complex1
1997 StandoutNobel
Mutations in mouse Aspm (abnormal spindle-like microcephaly associated) cause not only microcephaly but also major defects in the germline
2010 StandoutNobel
Hypoxic retinal Müller cells promote vascular permeability by HIF-1–dependent up-regulation of angiopoietin-like 4
2013 StandoutNobel
Abnormal phagocytosis by retinal pigmented epithelium that lacks myosin VIIa, the Usher syndrome 1B protein
2003
Nonviral Vectors for The Delivery of Small Interfering RNAs to The CNS
2010
Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy
2000
Rational Design of Bisphosphonate Lipid-like Materials for mRNA Delivery to the Bone Microenvironment
2022 StandoutNobel
Ciliary neurotrophic factor (CNTF) for human retinal degeneration: Phase I trial of CNTF delivered by encapsulated cell intraocular implants
2006
Transplantation of Neuroblastic Progenitor Cells as a Sheet Preserves and Restores Retinal Function
2005
Synaptic targeting domains of synapsin I revealed by transgenic expression in photoreceptor cells.
1994 StandoutNobel
Dynamic and quantitative Ca 2+ measurements using improved cameleons
1999 StandoutNobel
Conservation of sequence and structure flanking the mouse and human β-globin loci: The β-globin genes are embedded within an array of odorant receptor genes
1999 StandoutNobel
Computational design of a red fluorophore ligase for site-specific protein labeling in living cells
2014 StandoutNobel
Extracellular microRNAs in human circulation are associated with miRISC complexes that are accessible to anti-AGO2 antibody and can bind target mimic oligonucleotides
2020 StandoutNobel
Gene Therapy
2019
The proximal promoter of the mouse arrestin gene directs gene expression in photoreceptor cells and contains an evolutionarily conserved retinal factor-binding site.
1993
Functional and Structural Recovery of the Retina after Gene Therapy in the RPE65 Null Mutation Dog
2003
Works of Jean Bennett being referenced
Pigment epithelium-derived factor expression in the developing mouse eye.
2002
Localization of pigment epithelium derived factor (PEDF) in developing and adult human ocular tissues.
2001
Continuing Evaluation of Gene Therapy In The Rpe65 Mutant Dog
2002
bcl-2 Gene Therapy Exacerbates Excitotoxicity
1999
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
2005
Pharmacological Regulation of Protein Expression from Adeno-Associated Viral Vectors in the Eye
2002
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
2008
Novel AAV serotypes for improved ocular gene transfer
2008
Inhibition of Retinal Neovascularization by Intraocular Viral-Mediated Delivery of Anti-angiogenic Agents
2002
Long-Term Protection of Retinal Structure but Not Function Using RAAV.CNTF in Animal Models of Retinitis Pigmentosa
2001
Tie2Cre-mediated inactivation of plexinD1 results in congenital heart, vascular and skeletal defects
2008
Mutation of a nuclear receptor gene, NR2E3, causes enhanced S cone syndrome, a disorder of retinal cell fate
2000
Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa
2011
Humoral Response after Administration of E1-Deleted Adenoviruses: Immune Privilege of the Subretinal Space
1996
Spectrum of Mutations in the RPGR Gene That Are Identified in 20% of Families with X-Linked Retinitis Pigmentosa
1997
Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequence
2004
In Utero Gene Therapy Rescues Vision in a Murine Model of Congenital Blindness
2004
Dissecting the Axoneme Interactome
2005
Gene therapy for choroideremia: in vitro rescue mediated by recombinant adenovirus
2003
Unusual topography of bovine rhodopsin promoter-IacZ fusion gene expression in transgenic mouse retinas
1991
Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier
2016
Safety of Recombinant Adeno-Associated Virus Type 2–RPE65 Vector Delivered by Ocular Subretinal Injection
2006
Adenovirus-mediated delivery of rhodopsin-promoted bcl-2 results in a delay in photoreceptor cell death in the rd/rd mouse
1998
Light damage induced changes in mouse retinal gene expression
2004
Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting
2017
AAV-Mediated Delivery of Ciliary Neurotrophic Factor Prolongs Photoreceptor Survival in the Rhodopsin Knockout Mouse
2001
A Deviant Immune Response to Viral Proteins and Transgene Product Is Generated on Subretinal Administration of Adenovirus and Adeno-associated Virus
2002
Phenotypic rescue after adeno‐associated virus‐mediated delivery of 4‐sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI
2002
In Vivo Delivery of Recombinant Viruses to the Fetal Murine Cochlea: Transduction Characteristics and Long-Term Effects on Auditory Function
2006
Gene therapy restores vision in a canine model of childhood blindness
2001 Standout
Efficient Trans-Splicing in the Retina Expands the Utility of Adeno-Associated Virus as a Vector for Gene Therapy
2003
Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model.
2003
Lateral Connectivity and Contextual Interactions in Macaque Primary Visual Cortex
2002
Nonhuman Primate Models for Diabetic Ocular Neovascularization Using AAV2-Mediated Overexpression of Vascular Endothelial Growth Factor
2005
Disease expression of RP1 mutations causing autosomal dominant retinitis pigmentosa.
2000
Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV
1999
2001
Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success
2005
How long does gene therapy last? 4-year follow-up of phase 3 voretigene neparvovec trial in RPE65-associated LCA/inherited retinal disease
2019
Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey
2011
Impairment of the Transient Pupillary Light Reflex inRpe65−/−Mice and Humans with Leber Congenital Amaurosis
2004
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina
1999
Real-time, noninvasive in vivo assessment of adeno-associated virus-mediated retinal transduction.
1997
Cell-mediated immune response and stability of intraocular transgene expression after adenovirus-mediated delivery.
1997
Herpes Simplex Virus Type 1 Glycoprotein E Is Required for Axonal Localization of Capsid, Tegument, and Membrane Glycoproteins
2005
Adenovirus vector-mediated in vivo gene transfer into adult murine retina.
1994
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
2012