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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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Biology and therapy of inherited retinal degenerative disease: insights from mouse models

Disease models & mechanisms, 2015-02, Vol.8 (2), p.109-129

2015

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Genetic Rescue of X-Linked Retinoschisis Mouse ( Rs1 -/y ) Retina Induces Quiescence of the Retinal Microglial Inflammatory State Following AAV8- RS1 Gene Transfer and Identifies Gene Networks Underlying Retinal Recovery

Human gene therapy, 2021-07, Vol.32 (13-14), p.667-681

2021

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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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A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration

Human molecular genetics, 2015-07, Vol.24 (14), p.3956-3970

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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Subjective and Objective Screening Tests for Hydroxychloroquine Toxicity

Ophthalmology (Rochester, Minn.), 2015-02, Vol.122 (2), p.356-366

2015

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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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Analysis of Anatomic and Functional Measures in X-Linked Retinoschisis

Investigative ophthalmology & visual science, 2018-06, Vol.59 (7), p.2841-2847

2018

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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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Homozygosity Mapping and Genetic Analysis of Autosomal Recessive Retinal Dystrophies in 144 Consanguineous Pakistani Families

Investigative ophthalmology & visual science, 2017-04, Vol.58 (4), p.2218-2238

2017

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Rearing Light Intensity Affects Inner Retinal Pathology in a Mouse Model of X-Linked Retinoschisis but Does Not Alter Gene Therapy Outcome

Investigative ophthalmology & visual science, 2017-03, Vol.58 (3), p.1656-1664

2017

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XLRS Rat with Rs1 -/Y Exon-1-Del Shows Failure of Early Postnatal Outer Retina Development

Genes, 2022-10, Vol.13 (11), p.1995

2022

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Exome analysis identified a novel mutation in the RBP4 gene in a consanguineous pedigree with retinal dystrophy and developmental abnormalities

PloS one, 2012-11, Vol.7 (11), p.e50205-e50205

2012

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A Splice-Site Mutation in a Retina-Specific Exon of BBS8 Causes Nonsyndromic Retinitis Pigmentosa

American journal of human genetics, 2010-05, Vol.86 (5), p.805-812

2010

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OCT Intensity of the Region between Outer Retina Band 2 and Band 3 as a Biomarker for Retinal Degeneration and Therapy

Bioengineering (Basel), 2024-05, Vol.11 (5), p.449

2024

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Predominant Founder Effect among Recurrent Pathogenic Variants for an X-Linked Disorder

Genes, 2022-04, Vol.13 (4), p.675

2022

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Treatment with Isotretinoin Inhibits Lipofuscin Accumulation in a Mouse Model of Recessive Stargardt's Macular Degeneration

Proceedings of the National Academy of Sciences - PNAS, 2003-04, Vol.100 (8), p.4742-4747

2003

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CNTF and retina

Progress in retinal and eye research, 2012-03, Vol.31 (2), p.136-151

2012

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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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Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation

Gene therapy, 2022-08, Vol.29 (7-8), p.431-440

2022

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A Mutation in SLC24A1 Implicated in Autosomal-Recessive Congenital Stationary Night Blindness

American journal of human genetics, 2010-10, Vol.87 (4), p.523-531

2010

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Tissue engineering: NIH competition to create 'eye in a dish'

Nature (London), 2017-06, Vol.546 (7658), p.352-352

Sieving, Paul A

2017

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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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“Para-retinal” Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression

Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.422-427

2020

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

Biology and therapy of inherited retinal degenerative disease: insights from mouse models

Genetic Rescue of X-Linked Retinoschisis Mouse ( Rs1 -/y ) Retina Induces Quiescence of the Retinal Microglial Inflammatory State Following AAV8- RS1 Gene Transfer and Identifies Gene Networks Underlying Retinal Recovery

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

A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration

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

Subjective and Objective Screening Tests for Hydroxychloroquine Toxicity

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

Analysis of Anatomic and Functional Measures in X-Linked Retinoschisis

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

Homozygosity Mapping and Genetic Analysis of Autosomal Recessive Retinal Dystrophies in 144 Consanguineous Pakistani Families

Rearing Light Intensity Affects Inner Retinal Pathology in a Mouse Model of X-Linked Retinoschisis but Does Not Alter Gene Therapy Outcome

XLRS Rat with Rs1 -/Y Exon-1-Del Shows Failure of Early Postnatal Outer Retina Development

Exome analysis identified a novel mutation in the RBP4 gene in a consanguineous pedigree with retinal dystrophy and developmental abnormalities

A Splice-Site Mutation in a Retina-Specific Exon of BBS8 Causes Nonsyndromic Retinitis Pigmentosa

OCT Intensity of the Region between Outer Retina Band 2 and Band 3 as a Biomarker for Retinal Degeneration and Therapy

Predominant Founder Effect among Recurrent Pathogenic Variants for an X-Linked Disorder

Treatment with Isotretinoin Inhibits Lipofuscin Accumulation in a Mouse Model of Recessive Stargardt's Macular Degeneration

CNTF and retina

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

Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation

A Mutation in SLC24A1 Implicated in Autosomal-Recessive Congenital Stationary Night Blindness

Tissue engineering: NIH competition to create 'eye in a dish'

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

“Para-retinal” Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression

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