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Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Molecular therapy, 2018-09, Vol.26 (9), p.2282-2294

2018

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Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer

The Journal of clinical investigation, 2015-07, Vol.125 (7), p.2891-2903

2015

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Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function

Human gene therapy, 2016-05, Vol.27 (5), p.376-389

2016

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Retinal Structure and Gene Therapy Outcome in Retinoschisin-Deficient Mice Assessed by Spectral-Domain Optical Coherence Tomography

Investigative ophthalmology & visual science, 2016-07, Vol.57 (9), p.OCT277-OCT287

2016

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Ciliary Neurotrophic Factor (CNTF) for Human Retinal Degeneration: Phase I Trial of CNTF Delivered by Encapsulated Cell Intraocular Implants

Proceedings of the National Academy of Sciences - PNAS, 2006-03, Vol.103 (10), p.3896-3901

2006

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Trans-ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Gene Transduction after Intravitreal Vector Administration

Molecular therapy. Methods & clinical development, 2019-06, Vol.13, p.77-85

2019

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Photopic ERG Negative Response from Amacrine Cell Signaling in RCS Rat Retinal Degeneration

Investigative ophthalmology & visual science, 2008-01, Vol.49 (1), p.442-452

2008

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Regulation of Rod Phototransduction Machinery by Ciliary Neurotrophic Factor

The Journal of neuroscience, 2006-12, Vol.26 (52), p.13523-13530

2006

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Depleting Rac1 in mouse rod photoreceptors protects them from photo-oxidative stress without affecting their structure or function

Proceedings of the National Academy of Sciences - PNAS, 2009-06, Vol.106 (23), p.9397-9402

2009

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RanBP2 modulates Cox11 and hexokinase I activities and haploinsufficiency of RanBP2 causes deficits in glucose metabolism

PLoS genetics, 2006-10, Vol.2 (10), p.e177

2006

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Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice

Molecular therapy. Methods & clinical development, 2016-01, Vol.5 (C), p.16011-16011, Article 16011

2016

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Probing potassium channel function in vivo by intracellular delivery of antibodies in a rat model of retinal neurodegeneration

Proceedings of the National Academy of Sciences - PNAS, 2010-07, Vol.107 (28), p.12710-12715

2010

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Cone ERG Changes During Light Adaptation in Two All-Cone Mutant Mice: Implications for Rod-Cone Pathway Interactions

Investigative ophthalmology & visual science, 2019-08, Vol.60 (10), p.3680-3688

2019

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AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration

Gene therapy, 2019-09, Vol.26 (9), p.386-398

2019

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Retinopathy induced in mice by targeted disruption of the rhodopsin gene

Nature genetics, 1997-02, Vol.15 (2), p.216-219

1997

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Encapsulated Cell-Based Intraocular Delivery of Ciliary Neurotrophic Factor in Normal Rabbit: Dose-Dependent Effects on ERG and Retinal Histology

Investigative ophthalmology & visual science, 2004-07, Vol.45 (7), p.2420-2430

2004

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NADPH Oxidase Contributes to Photoreceptor Degeneration in Constitutively Active RAC1 Mice

Investigative ophthalmology & visual science, 2016-05, Vol.57 (6), p.2864-2875

2016

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Retinoschisin Gene Therapy and Natural History in the Rs1h-KO Mouse: Long-term Rescue from Retinal Degeneration

Investigative ophthalmology & visual science, 2007-08, Vol.48 (8), p.3837-3845

2007

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X-linked retinoschisis: RS1 mutation severity and age affect the ERG phenotype in a cohort of 68 affected male subjects

Investigative ophthalmology & visual science, 2011-11, Vol.52 (12), p.9250-9256

2011

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CNGB3-achromatopsia clinical trial with CNTF: diminished rod pathway responses with no evidence of improvement in cone function

Investigative ophthalmology & visual science, 2014-10, Vol.55 (10), p.6301-6308

2014

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Intravitreal Ciliary Neurotrophic Factor Transiently Improves Cone-Mediated Function in a CNGB3-/- Mouse Model of Achromatopsia

Investigative ophthalmology & visual science, 2015-10, Vol.56 (11), p.6810-6822

2015

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Loss of retinoschisin (RS1) cell surface protein in maturing mouse rod photoreceptors elevates the luminance threshold for light-driven translocation of transducin but not arrestin

The Journal of neuroscience, 2012-09, Vol.32 (38), p.13010-13021

2012

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Nrl is required for rod photoreceptor development

Nature genetics, 2001-12, Vol.29 (4), p.447-452

2001

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Synaptic Pathology in Retinoschisis Knockout (Rs1-/y) Mouse Retina and Modification by rAAV-Rs1 Gene Delivery

Investigative ophthalmology & visual science, 2008-08, Vol.49 (8), p.3677-3686

2008

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Convergence of Human Genetics and Animal Studies: Gene Therapy for X-Linked Retinoschisis

Cold Spring Harbor perspectives in medicine, 2015-08, Vol.5 (8), p.a017368-a017368

2015

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Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer

Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function

Retinal Structure and Gene Therapy Outcome in Retinoschisin-Deficient Mice Assessed by Spectral-Domain Optical Coherence Tomography

Ciliary Neurotrophic Factor (CNTF) for Human Retinal Degeneration: Phase I Trial of CNTF Delivered by Encapsulated Cell Intraocular Implants

Trans-ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Gene Transduction after Intravitreal Vector Administration

Photopic ERG Negative Response from Amacrine Cell Signaling in RCS Rat Retinal Degeneration

Regulation of Rod Phototransduction Machinery by Ciliary Neurotrophic Factor

Depleting Rac1 in mouse rod photoreceptors protects them from photo-oxidative stress without affecting their structure or function

RanBP2 modulates Cox11 and hexokinase I activities and haploinsufficiency of RanBP2 causes deficits in glucose metabolism

Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice

Probing potassium channel function in vivo by intracellular delivery of antibodies in a rat model of retinal neurodegeneration

Cone ERG Changes During Light Adaptation in Two All-Cone Mutant Mice: Implications for Rod-Cone Pathway Interactions

AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration

Retinopathy induced in mice by targeted disruption of the rhodopsin gene

Encapsulated Cell-Based Intraocular Delivery of Ciliary Neurotrophic Factor in Normal Rabbit: Dose-Dependent Effects on ERG and Retinal Histology

NADPH Oxidase Contributes to Photoreceptor Degeneration in Constitutively Active RAC1 Mice

Retinoschisin Gene Therapy and Natural History in the Rs1h-KO Mouse: Long-term Rescue from Retinal Degeneration

X-linked retinoschisis: RS1 mutation severity and age affect the ERG phenotype in a cohort of 68 affected male subjects

CNGB3-achromatopsia clinical trial with CNTF: diminished rod pathway responses with no evidence of improvement in cone function

Intravitreal Ciliary Neurotrophic Factor Transiently Improves Cone-Mediated Function in a CNGB3-/- Mouse Model of Achromatopsia

Loss of retinoschisin (RS1) cell surface protein in maturing mouse rod photoreceptors elevates the luminance threshold for light-driven translocation of transducin but not arrestin

Nrl is required for rod photoreceptor development

Synaptic Pathology in Retinoschisis Knockout (Rs1-/y) Mouse Retina and Modification by rAAV-Rs1 Gene Delivery

Convergence of Human Genetics and Animal Studies: Gene Therapy for X-Linked Retinoschisis

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