Jean Bennett

Q: What Schools Are Affiliated With Jean Bennett

Jean Bennett is affiliated with the following schools: University of California, Berkeley, Yale University, University of California, San Francisco, University of Pennsylvania, Harvard University

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Jean Bennett

Medical

#904

World Rank

#1167

Historical Rank

#306

USA Rank

Ophthalmology

#26

World Rank

#38

Historical Rank

USA Rank

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Medical

Jean Bennett's Degrees

Doctorate Medicine Harvard University
PhD Biochemistry University of California, San Francisco

Why Is Jean Bennett Influential?

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According to Wikipedia, Jean Bennett is the F. M. Kirby Professor of Ophthalmology in the Perelman School of Medicine at the University of Pennsylvania. Her research focuses on gene therapy for retinal diseases. Her laboratory developed the first FDA approved gene therapy for use in humans, which treats a rare form of blindness. She was elected a member of the National Academy of Sciences in 2022.

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Jean Bennett's Published Works

Number of citations in a given year to any of this author's works

Total number of citations to an author for the works they published in a given year. This highlights publication of the most important work(s) by the author

Published Works

Safety and efficacy of gene transfer for Leber's congenital amaurosis. (2008) (2022)
Gene therapy restores vision in a canine model of childhood blindness (2001) (1172)
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial (2017) (861)
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial (2009) (752)
Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration (2009) (551)
Lateral Connectivity and Contextual Interactions in Macaque Primary Visual Cortex (2002) (484)
Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model. (2003) (465)
Mutation of a nuclear receptor gene, NR2E3, causes enhanced S cone syndrome, a disorder of retinal cell fate (2000) (463)
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) (461)
Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. (2001) (384)
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness (2012) (384)
Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy (1996) (339)
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial (2016) (327)
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. (2013) (317)
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. (2008) (279)
Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success (2005) (267)
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. (1999) (256)
Adenovirus vector-mediated in vivo gene transfer into adult murine retina. (1994) (246)
Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequence. (2004) (237)
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. (2008) (235)
Long-term protection of retinal structure but not function using RAAV.CNTF in animal models of retinitis pigmentosa. (2001) (223)
Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies. (2010) (217)
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. (2006) (215)
In utero gene therapy rescues vision in a murine model of congenital blindness. (2004) (203)
Real-time, noninvasive in vivo assessment of adeno-associated virus-mediated retinal transduction. (1997) (198)
Novel AAV serotypes for improved ocular gene transfer (2008) (187)
AAV-mediated delivery of ciliary neurotrophic factor prolongs photoreceptor survival in the rhodopsin knockout mouse. (2001) (183)
Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey (2011) (180)
Progress in gene therapy for neurological disorders (2013) (172)
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials. (2019) (171)
Light damage induced changes in mouse retinal gene expression. (2004) (158)
Localization of pigment epithelium derived factor (PEDF) in developing and adult human ocular tissues. (2001) (154)
Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV (1999) (151)
Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter (2014) (147)
Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents. (2002) (141)
The human visual cortex responds to gene therapy-mediated recovery of retinal function. (2011) (141)
Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa (2011) (133)
Disease expression of RP1 mutations causing autosomal dominant retinitis pigmentosa. (2000) (131)
The Status of RPE65 Gene Therapy Trials: Safety and Efficacy. (2015) (130)
Immune response following intraocular delivery of recombinant viral vectors (2003) (124)
Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290 (2012) (124)
Deficiency of SPAG16L Causes Male Infertility Associated with Impaired Sperm Motility1 (2006) (120)
Adenovirus-mediated delivery of rhodopsin-promoted bcl-2 results in a delay in photoreceptor cell death in the rd/rd mouse (1998) (117)
ABCA4-associated retinal degenerations spare structure and function of the human parapapillary retina. (2005) (116)
In vivo gene transfer into murine corneal endothelial and trabecular meshwork cells. (1995) (115)
Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness (2010) (111)
Macular pigment and lutein supplementation in choroideremia. (2002) (107)
In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery. (2016) (107)
Cell-mediated immune response and stability of intraocular transgene expression after adenovirus-mediated delivery. (1997) (105)
Systemic rapamycin inhibits retinal and choroidal neovascularization in mice. (2003) (103)
In vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory function. (2006) (103)
Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosis. (2004) (102)
Mutations in RPGR and RP2 account for 15% of males with simplex retinal degenerative disease. (2012) (101)
Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration. (2004) (100)
High-resolution adaptive optics retinal imaging of cellular structure in choroideremia. (2014) (98)
Spectrum of mutations in the RPGR gene that are identified in 20% of families with X-linked retinitis pigmentosa. (1997) (97)
Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina. (2017) (97)
Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy. (2003) (96)
Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye. (2002) (90)
Increased expression of ceruloplasmin in the retina following photic injury. (2003) (87)
Natural History of the Central Structural Abnormalities in Choroideremia: A Prospective Cross-Sectional Study. (2017) (86)
Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward. (2017) (85)
AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models (2013) (84)
Immunology of AAV-Mediated Gene Transfer in the Eye (2013) (84)
AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina (2013) (83)
AAV2.7m8 is a powerful viral vector for inner ear gene therapy (2019) (83)
Retinal ganglion cell loss induced by acute optic neuritis in a relapsing model of multiple sclerosis (2006) (80)
Novel mobility test to assess functional vision in patients with inherited retinal dystrophies (2018) (80)
Basal exon skipping and genetic pleiotropy: A predictive model of disease pathogenesis (2015) (76)
Disruption of CEP290 microtubule/membrane-binding domains causes retinal degeneration. (2013) (74)
Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor. (2005) (63)
Dissecting the Axoneme Interactome (2005) (62)
Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier. (2016) (59)
Proapoptotic bcl-2 family members, Bax and Bak, are essential for developmental photoreceptor apoptosis. (2003) (59)
Humoral response after administration of E1-deleted adenoviruses: immune privilege of the subretinal space. (1996) (57)
Melatonin delays photoreceptor degeneration in the rds/rds mouse (2001) (57)
Gene therapy and retinitis pigmentosa: advances and future challenges (2001) (57)
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. (2007) (56)
Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates. (2005) (56)
Disease expression in Usher syndrome caused by VLGR1 gene mutation (USH2C) and comparison with USH2A phenotype. (2005) (55)
Additional transduction events after subretinal readministration of recombinant adeno-associated virus. (2000) (55)
Herpes Simplex Virus Type 1 Glycoprotein E Is Required for Axonal Localization of Capsid, Tegument, and Membrane Glycoproteins (2005) (55)
Adeno-associated viruses containing bFGF or BDNF are neuroprotective against excitotoxicity (2004) (54)
Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting (2017) (54)
A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus. (2002) (54)
Gene therapy for choroideremia: in vitro rescue mediated by recombinant adenovirus (2003) (53)
Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector. (2007) (50)
Oxygen distribution and vascular injury in the mouse eye measured by phosphorescence-lifetime imaging. (2004) (50)
Plasticity of the human visual system after retinal gene therapy in patients with Leber’s congenital amaurosis (2015) (50)
Delivery of Adeno-Associated Virus Vectors to the Fetal Retina: Impact of Viral Capsid Proteins on Retinal Neuronal Progenitor Transduction (2003) (49)
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy. (2020) (47)
Novel mutation in the TIMP3 gene causes Sorsby fundus dystrophy. (2002) (47)
Seeing the Light (2013) (46)
Pigment epithelium-derived factor expression in the developing mouse eye. (2002) (44)
The Pex1-G844D mouse: a model for mild human Zellweger spectrum disorder. (2014) (43)
Gene therapy and animal models for retinal disease. (2003) (43)
Gene therapy for ocular disease. (2000) (43)
Phenotypic rescue after adeno‐associated virus‐mediated delivery of 4‐sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI (2002) (42)
Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system. (2006) (42)
Gene therapy in ophthalmology: validation on cultured retinal cells and explants from postmortem human eyes. (2011) (41)
Gene therapy for color blindness. (2009) (40)
Adenovirus-mediated delivery of catalase to retinal pigment epithelial cells protects neighboring photoreceptors from photo-oxidative stress. (2004) (39)
Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 Years and 4 Years. (2021) (39)
Persistent fetal ocular vasculature in mice deficient in bax and bak. (2005) (39)
RDH12 Mutations Cause a Severe Retinal Degeneration With Relatively Spared Rod Function. (2018) (38)
The Thioredoxin Encoded by the Rod-Derived Cone Viability Factor Gene Protects Cone Photoreceptors Against Oxidative Stress. (2016) (36)
Cross-species comparison of in vivo reporter gene expression after recombinant adeno-associated virus-mediated retinal transduction. (2000) (35)
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV (2020) (35)
Adeno-associated virus mediated gene therapy for retinal degenerative diseases. (2011) (34)
Screening of a Large Cohort of Leber Congenital Amaurosis and Retinitis Pigmentosa Patients Identifies Novel LCA5 Mutations and New Genotype–Phenotype Correlations (2013) (34)
Thioredoxin rod-derived cone viability factor protects against photooxidative retinal damage. (2015) (34)
Use of induced pluripotent stem cell models to probe the pathogenesis of Choroideremia and to develop a potential treatment. (2018) (33)
SIRT1 and NRF2 Gene Transfer Mediate Distinct Neuroprotective Effects Upon Retinal Ganglion Cell Survival and Function in Experimental Optic Neuritis (2018) (32)
Progress in gene therapy for neurological disorders (2013) (31)
CEP290 and the primary cilium. (2014) (31)
Adeno‐associated virus 8‐mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models (2014) (31)
Gene therapy for Leber congenital amaurosis. (2004) (30)
Pharmacologically regulated gene expression in the retina following transduction with viral vectors (2001) (28)
Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients (2012) (25)
Preferential rod and cone photoreceptor abnormalities in heterozygotes with point mutations in the RDS gene. (1996) (25)
The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients. (2017) (23)
fMRI of Retina-Originated Phosphenes Experienced by Patients with Leber Congenital Amaurosis (2014) (23)
Nxnl2 splicing results in dual functions in neuronal cell survival and maintenance of cell integrity. (2012) (23)
Adeno-Associated Virus-Mediated Gene Transfer to Renal Tubule Cells via a Retrograde Ureteral Approach (2011) (23)
Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA (2018) (23)
Dark-rearing the rd10 mouse: implications for therapy. (2012) (22)
bcl-2 gene therapy exacerbates excitotoxicity. (1999) (21)
Commentary: an aye for eye gene therapy. (2006) (21)
Comparative AAV-eGFP Transgene Expression Using Vector Serotypes 1–9, 7m8, and 8b in Human Pluripotent Stem Cells, RPEs, and Human and Rat Cortical Neurons (2019) (20)
Prescreening whole exome sequencing results from patients with retinal degeneration for variants in genes associated with retinal degeneration (2017) (20)
Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho–/– mouse (2015) (20)
CHOROIDEREMIA: Retinal Degeneration With an Unmet Need. (2019) (19)
Strain‐dependent anterior segment neovascularization following intravitreal gene transfer of basic fibroblast growth factor (bFGF) (2001) (18)
The Status of RPE 65 Gene Therapy Trials : Safety and Efficacy (2015) (18)
Gene therapy for Leber congenital amaurosis. (2003) (17)
Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates (2019) (17)
Gene delivery to the retina: from mouse to man. (2012) (16)
Isolated maculopathy associated with biallelic CRB1 mutations (2017) (16)
Retinal progenitor cells--timing is everything. (2007) (16)
Adeno-associated virus encoding green fluorescent protein as a label for retinal pigment epithelium. (2003) (16)
Gene therapy for ocular neovascularization: a cure in sight. (2003) (16)
Depicting brighter possibilities for treating blindness (2019) (16)
Infection with adeno-associated virus may protect against excitotoxicity. (1999) (15)
Visual Function at the Atrophic Border in Choroideremia Assessed with Adaptive Optics Microperimetry. (2019) (15)
Using the NAFX to measure the effectiveness over time of gene therapy in canine LCA. (2009) (15)
Letter to the Editor: A Carboxy-terminal Truncation of 99 Amino Acids Resulting from a Novel Mutation (Arg555→stop) in theCHMGene leads to Choroideremia (1997) (14)
Effect of over-expression of pigment epithelium derived factor (PEDF) on developing retinal vasculature in the mouse. (2003) (14)
How long does gene therapy last? 4-year follow-up of phase 3 voretigene neparvovec trial in RPE65-associated LCA/inherited retinal disease (2019) (13)
An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications (2015) (13)
Comparing Clinical Perimetry and Population Receptive Field Measures in Patients with Choroideremia (2018) (13)
Imaging oxygen pressure in the retina of the mouse eye. (2005) (13)
Disease Expression of RP 1 Mutations Causing Autosomal Dominant Retinitis Pigmentosa (2000) (12)
The distribution, concentration, and toxicity of enhanced green fluorescent protein in retinal cells after genomic or somatic (virus-mediated) gene transfer. (2005) (12)
Gene therapy for retinitis pigmentosa. (2000) (11)
Utility of In Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity Before Gene Therapy. (2019) (11)
Imaging oxygen pressure in the rodent retina by phosphorescence lifetime. (2006) (10)
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness. (2018) (10)
A carboxy-terminal truncation of 99 amino acids resulting from a novel mutation (Arg555-->stop) in the CHM gene leads to choroideremia. (1997) (10)
Correction: AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina (2013) (10)
CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters (2019) (10)
Compensatory Cross-Modal Plasticity Persists After Sight Restoration (2020) (9)
Novel mathematical algorithm for pupillometric data analysis (2014) (9)
Spatial and temporal expression patterns of the choroideremia gene in the mouse retina. (2005) (8)
AAV2-hCHM Subretinal Delivery to the Macula in Choroideremia: 2 year Results of an Ongoing Phase I/II Gene Therapy Trial (2019) (8)
Detailed retinal phenotype of Boucher-Neuhäuser syndrome associated with mutations in PNPLA6 mimicking choroideremia (2019) (8)
Treatment Potential for LCA5-Associated Leber Congenital Amaurosis (2020) (8)
Year 2 results for a phase 3 trial of voretigene neparvovec in biallelic RPE65-mediated inherited retinal disease (2017) (8)
A CRISPR view of the 2020 Nobel Prize in Chemistry. (2020) (8)
Gene Therapy to Abolish Congenital Nystagmus in RPE65-Deficient Canines (2003) (7)
Differential targeting of feline photoreceptors by recombinant adeno-associated viral vectors: implications for preclinical gene therapy trials (2014) (7)
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1 (2021) (7)
Identification of a novel pathogenic missense mutation in PRPF31 using whole exome sequencing: a case report (2018) (6)
Institutional Conflict of Interest. (2016) (6)
Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation–associated retinal dystrophy (2018) (6)
Cross-adaptation combined with TMS reveals a functional overlap between vision and imagery in the early visual cortex (2014) (6)
Switching on the lights: the use of optogenetics to advance retinal gene therapy. (2011) (6)
Bardet-Biedl syndrome-7 (BBS7) shows treatment potential and a cone-rod dystrophy phenotype that recapitulates the non-human primate model (2021) (6)
Continuing Evaluation of Gene Therapy In The Rpe65 Mutant Dog (2002) (6)
A Virtual Reality Orientation and Mobility Test for Inherited Retinal Degenerations: Testing a Proof-of-Concept After Gene Therapy (2021) (6)
AKT3 Gene Transfer Promotes Anabolic Reprogramming and Photoreceptor Neuroprotection in a Pre-clinical Model of Retinitis Pigmentosa. (2019) (6)
Evaluation of Ocular Gene Therapy in an Italian Patient Affected by Congenital Leber Amaurosis Type 2 Treated in Both Eyes. (2016) (5)
Strategies for delivery of rod-derived cone viability factor. (2005) (5)
AAV2-hCHM Subretinal Delivery to the Macula in Choroideremia: Preliminary Six Month Safety Results of an Ongoing Phase I/II Gene Therapy Trial (2017) (5)
AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder (2021) (5)
Exploiting metabolic and antioxidant pathways to maintain vision in blinding disease. (2015) (5)
Retinas in a Dish Peek into Inherited Retinal Degeneration. (2016) (4)
My career path for developing gene therapy for blinding diseases: the importance of mentors, collaborators, and opportunities. (2014) (4)
Stem cells set their sights on retinitis pigmentosa (2013) (4)
Müller cell transduction by AAV2 in normal and degenerative retinas. (2003) (4)
Imaging Retinal Structure in Patients and Carriers of Choroideremia (2012) (4)
Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models (2015) (4)
Application of adenoviral vectors. Analysis of eye development. (2000) (4)
On variants and disease-causing mutations: Case studies of a SEMA4A variant identified in inherited blindness (2018) (4)
Correlation of multi-luminance mobility testing with visual function tests in a phase 3 trial of voretigene neparvovec for biallelic RPE65-mediated inherited retinal disease (2017) (3)
Heat-inducible AAV-mediated Gene Delivery in RPE Cells (2003) (3)
Safety of the Subretinal Delivery of RGX-314 AAV8-anti-VEGF Fab Gene Therapy in NHP: Retinal Structure Over One Year (2018) (3)
Comparative study of mammalian phototransduction in vivo: a prelude to preclinical treatment trials in retinal degenerations (1999) (3)
AAV2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial. (2022) (3)
Restoration of Vision and Retinal Responses After Adeno-Associated Virus–Mediated Optogenetic Therapy in Blind Dogs (2022) (3)
Erratum: Progress in gene therapy for neurological disorders (Nature Reviews Neurology (2013) 9 (277-291) DOI: 10.1038/nrneurol.2013.56) (2013) (3)
Cone And Rod Transduction With Alternative AAV Serotypes In The Macula Of Non-human Primates (2011) (3)
Natural History of Retinal Function and Structure in the Rpe65-/- Murine Model of Leber Congenital Amaurosis (2003) (3)
Visual Function Questionnaire Responses in the Voretigene Neparvovec Phase 3 Trial (2019) (3)
Results of phase III clinical trial subretinal gene therapy for RPE65-mediated Leber congenital amaurosis (LCA) (2016) (3)
Algorithm for pupillometric data analysis (2010) (3)
RNA Splicing Factor Mutations That Cause Retinitis Pigmentosa Result in Circadian Dysregulation (2019) (3)
Cellular-scale assessment of visual function in Choroideremia (2018) (3)
The Signalling Role of the αvβ5-Integrin Can Impact the Efficacy of AAV in Retinal Gene Therapy (2012) (3)
The Bionic Retina: A Small Molecule with Big Potential for Visual Restoration (2012) (3)
Gene Therapy for Leber’s Congenital Amaurosis Due to RPE65 Mutations (2015) (3)
Three-year update for the phase 3 voretigene neparvovec study in biallelic RPE65 mutation–associated inherited retinal disease (2018) (2)
An Optimized Adeno-Associated Virus (AAV) for Treatment of Leber Congenital Amaurosis (LCA-RPE65) (2007) (2)
Chapter 34 – Gene Therapy for Retinal Disease (2013) (2)
Adeno-associated Delivery of TIMP-3 Inhibits Experimental Choroidal Neovascularization (2003) (2)
Externally Regulated AAV-Mediated Delivery of PEDF Ameliorates the OIR Phenotype (2008) (2)
Blinded by the light: a nonhuman primate model of achromatopsia. (2019) (2)
In vivo Non–Viral Gene Transfer to Retinal Ganglion Cells (2006) (2)
Short-term Assessment of Subfoveal Injection of Adeno-Associated Virus-Mediated hCHM Gene Augmentation in Choroideremia Using Adaptive Optics Ophthalmoscopy. (2022) (2)
Short-term assessment of subfoveal injection of AAV2-hCHM gene augmentation in choroideremia using adaptive optics ophthalmoscopy (2021) (2)
Year 3 results and age-stratified analyses for a phase 3 trial of voretigene neparvovec in RPE65 mutation-associated inherited retinal disease (2018) (2)
Improving the Quantification of the Lateral Geniculate Nucleus in Magnetic Resonance Imaging Using a Novel 3D-Edge Enhancement Technique (2021) (2)
89 Five-year update for the Phase III voretigene neparvovec study in biallelic RPE65 mutation–associated inherited retinal disease (2021) (2)
Generation of transgenic mice for studies of ocular development and disease. (2001) (2)
Promising first steps in gene therapy for choroideremia. (2014) (2)
AAV-Mediated Retinal Transfer Of Anti-angiogenic Genes In A Murine Model Of Retinopathy Of Prematurity (ROP) (2002) (2)
Natural History of the Central Structural Abnormalities in Choroideremia: Insights from a Cross-sectional Study (2016) (2)
In Vivo RNA Interference in the Murine RPE (2003) (2)
Retinal structure - function comparison through adaptive optics imaging reveals disease pathogenesis in choroideremia (2014) (1)
The ABCs of AMD (1997) (1)
AAV8BP2 and AAV8 transduce the mammalian cochlear lateral wall and endolymphatic sac with high efficiency (2022) (1)
The thioredoxin RdCVFL protects against photo-oxidative retinal damage (2015) (1)
Safety of subretinal delivery of RGX-314 (AAV8-anti-VEGF Fab) in the non-human primate as assessed by full-field ERG (2018) (1)
Ciliary Neurotrophic Factor (CNTF) Delivered by Adeno-associated Viruses (AAV) Leads to Photoreceptor Loss and Impairs Retinal Function in Wild-type Rats (2003) (1)
Subfoveal gene augmentation therapy for choroideremia: One-year results from a Phase I/II trial of AAV2-hCHM (2019) (1)
Adaptive optics imaging of ABCA4 Retinal Degeneration (2014) (1)
Gene Therapy of a Mouse Model of Stargardt's Disease Using Adeno-Associated Viral Vectors Packaging Large Genes (2008) (1)
Erratum: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models (PLOS ONE (2015) 10:6 (e0129982) (doi:10.1371/journal.pone.0129982)) (2015) (1)
Bax and Bak are Essential for Developmental Apoptosis of Ocular Fetal Vasculature and INL Photoreceptors (2002) (1)
218. Nonhuman Primate Models for Retinal and Choroidal Neovascularization Using AAV2-Mediated Overexpression of Vascular Endothelial Growth Factor (2004) (1)
Screening of a Large Cohort of Leber Congenital Amaurosis and Retinitis Pigmentosa Patients Identifies Novel LCA5 Mutations and New Genotype–Phenotype Correlations (2014) (1)
Distinct profiles of abnormal ganglion cell activity in two forms of Leber’s congenital amaurosis (LCA): Implications for therapy (2014) (1)
Adeno_Associated Virus (AAV)-Mediated Transduction of the Macula in Non-Human Primates (NHPs): Cell-Specificity, Stability and Safety of Transduction After Subretinal Delivery of AAV2 and AAV2/8 Vectors (2007) (1)
Structural Findings in Carriers and Affected Individuals with Choroideremia - an Optical Coherence Tomography Study (2014) (1)
185. Safety Study by Validated Immunoassays in a Phase III Study of Subjects with Inherited Retinal Dystrophy Due to Mutations in the Gene Encoding Human Retinal Pigment Epithelium-Specific Protein 65 (RPE65) Injected with Adeno-Associated Viral Vectors (2016) (1)
Brain Pathways Enabling Vision in LCA Patients Before and After Gene Therapy (2016) (1)
Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients (2012) (1)
Light Avoidance in Mice May Be not Related to Photoreceptor-Driven Input. (2016) (1)
Adaptive Optics Scanning Laser Ophthalmoscopy and High Resolution Imaging in Autosomal Dominant Retinitis Pigmentosa Caused by a Novel PRPF31 Nonsense Mutation (2013) (1)
Adaptive Optics Scanning Laser Ophthalmoscopy and High Resolution Imaging in Autosomal Dominant Retinitis Pigmentosa Caused by a Novel PRPF31 Nonsense Mutation (2013) (1)
Protocols for Visually Guided Navigation Assessment of Efficacy of Retina-Directed Cell or Gene Therapy in Canines (2017) (1)
Gene Therapy for Childhood Onset Blindness (2003) (1)
Normal parameters of the full field ERG recorded with bipolar electrodes in Cynomolgus Macaque (Macaque fascicularis) (2017) (1)
Refining surgical techniques for efficient posterior semicircular canal gene delivery in the adult mammalian inner ear with minimal hearing loss (2021) (1)
AAV-mediated Delivery of Rod-derived Cone Viability Factor in a Mouse Model of Retinal Degeneration (2011) (1)
Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl. (2022) (1)
EGFP levels and toxicity in transgenic and virus injected animals. (2004) (1)
Cone Photoreceptor Inner and Outer Segment Mosaic Abnormalities in Choroideremia (2016) (1)
553. Comparision of AAV Serotypes for Gene Delivery to iPSC Derived RPE (2016) (1)
Mobility testing validation study - Using a novel, standardized mobility test to evaluate functional vision in patients with inherited retinal degeneration (2015) (1)
Cellular Specificity of Expression After Subretinal Injection of Fetal Murine Retina Is Dependent on Aav Dose (2008) (0)
The utility of AAV2/rh10 for gene delivery to murine photoreceptors (2014) (0)
fMRI of Recovered Cortical Visual Responses Following Gene Therapy in Dogs (2005) (0)
Comparison of dual AAV vector approaches for large gene delivery to the retina (2014) (0)
Expression of halorhodopsin in bipolar cells can restore ganglion cell light responses in a large animal (canine) model of cone-rod dystrophy. (2014) (0)
Progress in gene therapy for neurological (2013) (0)
Somatic in utero Gene Delivery to E14 Nrl-/- Photoreceptors Induces Rod Specific Protein Expression (2007) (0)
Flash Pupillometry as a Tool for High Sensitivity Non-invasive Testing of the Retinal Function in Mice (2011) (0)
Structural and Functional Abnormalities in Leber Congenital Amaurosis Caused by Mutations in RDH12: A Short-Term Longitudinal Study (2019) (0)
499. Retinal Gene Therapy for Peroxisome Biogenesis Disorders (2015) (0)
A High Content Screening Platform for the Measurement of Trophic Factors in Rodent Models of Retinitis Pigmentosa (2009) (0)
Suppression of Ocular Neovascularizatoin With Lentivirus–Mediated Delivery of HIF1 Short Hairpin RNA (2006) (0)
AAV-mediated Gene Correction in a Mutant Transgenic LacZ Mouse (2003) (0)
Effects of Retinal Gene Therapy on Auditory-Visual Cross Modal Plasticity: Does Re-Established Vision Kick Auditory Activity out of the Occipital Lobe? (2017) (0)
Plasticity of the Human Visual Pathways Formed by Ocular Gene Therapy (2014) (0)
Nrl Is Sufficient for the Differentiation of Functional Rods (2005) (0)
Plasticity of the Human Visual System in Response to Retinal Gene Therapy in a Group of Patients with Leber’s Congenital Amaurosis (2015) (0)
Ocular gene therapy: Clinical successes and lessons learned (2016) (0)
Adenovirus–mediated delivery of catalase protects the mouse retina in vivo and RPE cells in vitro from oxidative stress induced cell death (2004) (0)
Semi masked analysis reduces bias in whole exome sequencing analysis (2017) (0)
In Vivo Gene Transfer by Electroporation to Adult C57Bl6 Mouse Retina (2004) (0)
Corrigendum to “Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho–/– mouse” (2016) (0)
splicing results in dual functions in neuronal cell survival and Nxnl 2 maintenance of cell integrity (2013) (0)
Aav7 viral vectors for targeted delivery of rpe cells (2009) (0)
Adenoviral-VEGF Induced Corneal and Iris Neovascularization in a Mouse (2002) (0)
Benzyl Alcohol–Free Triamcinolone Acetonide: Retinal Toxicity and Modulation of Vascular Endothelial Growth Factor (2005) (0)
S&R Mol Ther 2011 (2013) (0)
Rapamycin-inducible Secretion of a Therapeutic Protein in the Eye After Transduction With Viral Vectors (2002) (0)
VEGF Directed siRNA Inhibits Hypoxia Induced VEGF Upregulation In Vitro (2003) (0)
Early Structural and Functional Abnormalities in Leber Congenital Amaurosis Caused by Mutations in RDH12 (2018) (0)
Oxygen Measurements in the Retina Using Phosphorescence Lifetime Imaging (2005) (0)
AAV Mediated Gene Transfer Restores Retinal and Visual Function in Lebercilin-/- (LCA5) Mice (2013) (0)
T Cell Predominant Response to AAV-Spike Protects hACE2 Mice from SARS-CoV-2 Pneumonia (2021) (0)
Retina RDH 12 Mutations Cause a Severe Retinal Degeneration With Relatively Spared Rod Function (2018) (0)
Motion-selective areas V5/MT and MST appear resistant to deterioration in choroideremia (2023) (0)
Effects of RPR65 Gene Therapy on Nystagmus in Two Canine Littermates: Evidence of a Critical Period for Ocular Motor System (OMS) Calibration (2006) (0)
270. Optogenetic Activation of Remnant Cone Cells Can Restore Ganglion Cell Light Responses in a Canine Model of Retinitis Pigmentosa (2016) (0)
Antisense oligonucleotide delivery is an effective therapeutic approach for CEP290-associated LCA (2016) (0)
135. AAV-Mediated Gene Delivery To Evaluate the Biology of an Inherited Macular Degeneration (2005) (0)
AAV-Mediated Delivery of Rhodopsin to the Rhodopsin Knockout Mouse (rho-/-) (2002) (0)
Co–Localization of EFEMP1 and TIMP3 in the Developing and Mature Mouse Retina (2005) (0)
Basel exon skipping - a novel model of disease pathogenesis to explain CEP290-associated retinal degeneration and related ciliopathies (2014) (0)
488. Adeno-Associated Viral Serotype 2/1 Is a Highly Efficient Vector for In Utero Gene Transfer to Hair Cell Precursors (2006) (0)
Automated cone identification in adaptive optics retinal images of Choroideremia using a convolutional neural network (2019) (0)
AAV2.7m8 is a powerful viral vector for inner ear gene therapy (2019) (0)
Long Term Outcome Of Birdshot Chorioretinopathy Treated By Fluocinolone Acetonide Intravitreal Implant (Retisert®): A Case Series Report (2011) (0)
Induced pluripotent stem cells as a model To test gene augmentation therapy for Choroideremia (2013) (0)
Blocking RPE Transduction Following Sub-Retinal Injection of AAV Enhances Photoreceptor Targeted Gene Transfer (2010) (0)
881. Safety, Efficacy and Biodistribution of Recombinant AAV2-RPE65 Vector Delivered by Ocular Subretinal Injection (2006) (0)
556. Comparative In Vitro Transduction Efficiency of AAV Vector Serotypes 1-9 in Different Cellular Models (2016) (0)
Loss of Lebercilin causes dysregulation of RPE maturation and ciliary function in cellular and animal models (2018) (0)
Author Correction: An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications (2018) (0)
Long-term Safety and Efficacy of Aav-mediated Gene Transfer to the Retina of Patients Affected With Leber’s Congenital Amaurosis Due to Rpe65 Mutations (2009) (0)
Adenoviral-VEGF Induced Pre-retinal and Optic Nerve Neovascularization in a Mouse. (2002) (0)
Posterior Semicircular Canal Gene Delivery in the Adult Mammalian Inner Ear (2021) (0)
rAAV-mediated gene augmentation improves retinal and visual function and retinal structure in a mouse model for LCA5 (2016) (0)
Impact of Single Amino Acid Modifications of Adeno-Associated Virus (AAV) Capsid and Viral Dose on Transduction Patterns After Transuterine Subretinal Delivery of AAV Vectors to the Fetal Retina (2009) (0)
Trans-Splicing AAV Vector System Expands the Packaging Capacity Of AAV Gene Therapy Vectors for Delivery of Large Transgenes to the Retina (2002) (0)
Author Correction: An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications (2018) (0)
From DNA through FDA and EMA: Development of the first approved gene therapy for an inherited blinding disease (2019) (0)
Current gene therapy trials for inherited retinal disorders (2016) (0)
Nrl is a Master Regulator of Rod Photoreceptor Determination (2006) (0)
Delay of Rod Dark Adaptation Correlates with the Extent of Local Disease in ABCA4-associated Retinal Degenerations (2003) (0)
In Utero AAV Vector-Mediated Gene Delivery To The Retina (2002) (0)
Adeno-Catalase Protects the Balb/c Mouse Retina from Light-Damage (2003) (0)
Recombinant Adeno-Associated Virus (rAAV) Is Able to Transduce Retinal Muller Cells (2002) (0)
Correction (1999) (0)
Impaired Vision and Olfaction in Mice Lacking RdCVF2 (2009) (0)
Non-Viral Mediated in vivo Gene Delivery Facilitates Visual Recovery in Murine Model of Retinal Degeneration (2010) (0)
Subretinal AAV2/8 Is More Efficient At Targeting The Outer Reina Of The Cat Than AAV2/5 (2011) (0)
Induced Pluripotent Stem Cells as a Model To Test Gene Augmentation Therapy for Choroideremia [Conference abstract] (2013) (0)
Rpe65 gene therapy restores precise visual responses in vitro to many retinal ganglion cells in Rpe65-/- mice (2013) (0)
Parapapillary Retina Is Spared Retinal Degeneration in Human ABCA4 Disease (2006) (0)
Analysis of Photic Injury -induced Gene Expression by Microarray in the Rodent Retina (2002) (0)
Circadian Effects on Retinal Morphology: Relevance to Gene Transfer (2005) (0)
Baseline Fundus Autofluorescence Patterns In Leber Congenital Amaurosis (LCA) Patients With RPE65 Or LRAT Mutations (2011) (0)
189. Therapeutic Gene Transfer as a Treatment Option for Age-Related Macular Degeneration (2016) (0)
Modeling CRB1-associated LCA Using Patient-Specific Retinal Progenitor Cells (2018) (0)
Retinal Dystrophy New Early Signs Detection Towards Vision Improvement: A Case Presentation (2018) (0)
The Identification of Mutations in the REP–1 Promoter in a Patient with Choroideremia. (2004) (0)
Overview of Retinal Gene Therapy: Current Status and Future Challenges. (2022) (0)
AB037. rAAV mediated PEX1 gene augmentation improves visual function in a mouse model for Zellweger spectrum disorder (2018) (0)
Gene Targeting to Murine Retinal Bipolar Cells (2009) (0)
Impairment of Pupillary Light Responses in Murine Models of Retinal Degeneration (2010) (0)
Expression and Regulation of the Choroideremia Gene, Rep-1 (2003) (0)
Analysis of Inner Retinal Changes in Eyes of Rpe65–/– Mice Treated With Lentivirus–hRPE65 (2006) (0)
VEGF Directed siRNA Inhibits Experimental Choroidal Neovascularization (2003) (0)
Retinal Oxygen Imaging in Mice by Phosphorescence Lifetime (2004) (0)
High Throughput Behavioral Estimates of Visual Thresholds in Mice in a Watermaze with Cued Escape Platform (2013) (0)
Intravitreal injection of AAV7m8.hLCA5 restores photoreceptor function in Lca5-/- mice to nearly WT levels. (2017) (0)
Determination of Retinal Thermal Dosimetry from Transpupillary Thermotherapy (2002) (0)
Gene Replacement Therapy for Choroideremia (2011) (0)
Bax and Bak Are Essential, Overlapping Mediators of Apoptotic Developmental Regression of the Hyaloid Vasculature and of Retinal Neurons Including Photoreceptors (2003) (0)
Safety and Efficacy of Retinal Optogenetic Therapy in Canine Models (2013) (0)
Sequence of lipofuscin accumulation, retinoid cycle slowing and retinal degeneration in human ABCA4 disease (2004) (0)
886. AAV-Mediated Somatic Gene Transfer as an Approach To Delineate Pathogenic Mechanisms in an Autosomal Dominant Blindness Disorder Resembling Age-Related Macular Degeneration (AMD) (2006) (0)
Optimization and Validation of a Virtual Reality Orientation and Mobility Test for Inherited Retinal Degenerations (2023) (0)
Development of an Autogenous Transgene Regulation System for Ocular and Systemic Applications (2015) (0)
Assessing the tropism of adeno-associated virus serotypes in the primate retina for gene therapy (2015) (0)
Quantification of EGFP Expression in Living Cells of the Mouse Retina and Parallel Electrophysiological Assessment of Cell Health (2005) (0)
496. AAV-Mediated, Somatic Gene Transfer to RPE Cells in Order To Study the Pathogenesis of Macular Degeneration: Proteomic and Genomic Analysis (2006) (0)
Distribution of Fibulin 5 in Bruch’S Membrane and Defects Due to Missense Variations: Pathological Implications for Age–Related Macular Degeneration (2006) (0)
Trafficking of an Adeno-Associated Virus Variant in the Retina (2012) (0)
Circadian Pacemakers and the Cone Paraboloid (2005) (0)
Neuroplasticity of the Lateral Geniculate Nucleus in Response to Retinal Gene Therapy in a Group of Patients with RPE65 Mutations (2022) (0)
Preserved Island of Macular Retinal Structure in a Patient with Advanced RPE65-related Leber Congenital Amaurosis (2011) (0)
Subretinal delivery of RGX-314 AAV8-anti-VEGF Fab gene therapy in NHP (2017) (0)
Reliability of Counting Cone Inner Segments in Choroideremia (2017) (0)
Progress and Prospects in Ocular Gene Therapy (2008) (0)
Restoration of Pupillary Light Response in a Murine Model of Retinitis Pigmentosa Following AAV Mediated Gene Transfer (2010) (0)

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